Clinical Trial Results

Review results of recent St. Jude clinical trials

Clinical trials are a type of scientific research. They can help scientists learn about diseases and treatments in people. Patients who take part in our clinical trials, and their families, should have access to the results of that research. 

Research often takes a long time, so many years may pass before results are known. 

Learn more about taking part in clinical research >

Study results are often published in scientific and medical journals. St. Jude or another organization may also publish a notice about the results. If your clinical trial is not listed here, the study results may not yet be available. Be sure and check back later. After study results are published, the summaries will be posted here.

We offer our patients a simple summary that shows what we learned from the study. You can also ask your doctor or research participant advocate about the study. To reach the advocate, call 901-595-4644. If you are outside the Memphis area, dial 1-866-JUDE IRB (1-866-583-3472).

Each summary includes the name of the study, why the study was done, when the study took place and what we learned. It also gives details on the next research steps, how the study affected your child and where to learn more.

View recent clinical trial results below:

Blood Disorder Clinical Trials

  1. Results summary of St. Jude clinical trial:

    Pilot Study of Non-Invasive Assessment of Hepatic And Myocardial Iron Through T2* Magnet Resonance Imaging (MRI) In Patients With Iron Overload (MRIRON)

    Why was this study done?

    Iron overload is the buildup of iron in the body as a result of many blood transfusions. If iron builds up in important organs such as the heart and the liver, these organs may not work well. The iron in the body needs to be measured to figure out if it is time to begin a treatment to get rid of the iron from the body. Researchers wanted to find new ways to measure iron overload. This is usually done with a liver biopsy. But that procedure has risks that include pain, bleeding and infection. If we can measure iron overload by another way without a biopsy that would be important for patients.

    In this study, we used MRI imaging (magnetic resonance imaging, a way to obtain pictures of internal organs) and compare it to a liver biopsy to measure iron overload. T2* MRI has shown promise as a test to replace liver biopsy for this purpose. We wanted to compare the results of the T2* MRI with the results of the same patient’s liver biopsy.

    The study’s main goals were to:

    • Compare T2* MRI and biopsy measurements of iron in the liver
    • Compare T2* MRI and biopsy measurements of iron in the heart
    • Compare T2* MRI of the liver and the heart to the ferritin tests we do in the blood
    • Find out if a stronger MRI machine (3 Tesla MRI scanner) is better than the traditional MRI machine (1.5 Tesla MRI scanner)
    • Find out if the MRI can measure how well the heart works, compared with other heart tests (echo, EKG, MUGA scan)
    • Find out if new MRI tools (called “dark blood” and phantom) can help measure the iron in the heart and liver
    • Find out if increased pressure in the lungs can be linked to iron overload in the heart
    • Find out if genes in the liver are important in iron buildup

    When was this study done?

    The study opened in October 2005 and closed in June 2008.

    What did the study consist of?

    • MRI T2* and T2 of the liver and the heart
    •  Optional T2* liver scan on a 3 Tesla MRI (after the 1.5 Tesla MRI exam)
    • Blood tests
    • Liver biopsy
    • Heart tests

    What did we learn from this study?

    Iron overload and the heart: Diastolic dysfunction is a disorder that occurs when the chambers in the lower part of the heart do not work correctly. We learned that this problem is not caused by transfusions or iron deposits in the heart. Instead, it’s likely caused by sickle cell disease itself.

    Iron overload and the liver: In patients who receive many transfusions, the amount of liver damage depends on the rate and pattern of iron deposits. We learned more about these patterns. We found out that chelation therapy (a process to remove metals like iron from the body) may be underused and that mutations in certain genes, such as GSTM-1, may increase the iron accumulation in the liver by impairing how chelation medications work. We also learned that liver damage related to transfusions may begin earlier than we knew before.

    MRI vs. liver biopsy: We found strong evidence that an MRI T2* can predict iron content in the liver without the patient having to undergo a liver biopsy.

    What are the next research steps as a result of this study?

    Scientists will continue to research the genetic and disease-related processes that affect iron buildup.

    How does this study affect my child?

    Every individual with sickle cell disease should receive life-long follow-up care. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Ventricular diastolic dysfunction in sickle cell anemia is common but not associated with myocardial iron deposition. Hankins JS, McCarville MB, Hillenbrand CM, Loeffler RB, Ware RE, Song R, Smeltzer MP, Joshi V. Pediatr Blood Cancer. 2010 Sep;55(3):495-500.
    https://www.ncbi.nlm.nih.gov/pubmed/20658621

    Patterns of liver iron accumulation in patients with sickle cell disease and thalassemia with iron overload. Hankins JS, Smeltzer MP, McCarville MB, Aygun B, Hillenbrand CM, Ware RE, Onciu M. Eur J Haematol. 2010 Jul;85(1):51-7.
    https://www.ncbi.nlm.nih.gov/pubmed/20374273

    R2* magnetic resonance imaging of the liver in patients with iron overload. Hankins JS, McCarville MB, Loeffler RB, Smeltzer MP, Onciu M, Hoffer FA, Li CS, Wang WC, Ware RE, Hillenbrand CM. Blood. 2009 May 14;113(20):4853-5.
    https://www.ncbi.nlm.nih.gov/pubmed/19264677

    Microarray analysis of liver gene expression in iron overloaded patients with sickle cell anemia and beta-thalassemia. Flanagan JM, Steward S, Hankins JS, Howard TM, Neale G, Ware RE. Am J Hematol 2009 Jun;84(6):328-34.
    https://www.ncbi.nlm.nih.gov/pubmed/19384939

    GSTM1 and Liver Iron Content in Children with Sickle Cell Anemia and Iron Overload. Puri L, Flanagan JM, Kang G, Ding J, Bi W, McCarville BM, Loeffler RB, Tipirneni-Sajja A, Villavicencio M, Crews KR, Hillenbrand CM6 Hankins JS. J Clin Med. 2019 Nov 5;8(11).
    https://www.ncbi.nlm.nih.gov/pubmed/31694285

Brain Tumor Clinical Trials

  1. Results summary of St. Jude clinical trial:

    A safety/feasibility trial of the addition of the humanized anti-GD2 antibody (HU14.18K322A) with and without natural killer cells to chemotherapy in children and adolescents with recurrent/refractory neuroblastoma (GD2NK)

    Why was this study done?

    GD2NK used an antibody called hu14.18K322A and natural killer (NK) cells in patients who have neuroblastoma that has come back or is hard to treat. This treatment was given along with chemotherapy (chemo).

    Our immune system looks for and attacks cells that it sees as harmful, such as bacteria, viruses and cancer cells. Antibodies are part of the immune system. Hu14.18K322A is a monoclonal antibody, a protein made in a lab. We designed hu14.18K322A to bind to cancer cells that contain or “express” a molecule called an anti-GD2 antigen. Almost all neuroblastoma cells express this antigen. When hu14.18K322A binds to these types of cancer cells, it tells the immune system to attack and kill the cancer cells without harming nearby healthy cells.

    NK cells are also special cells in our immune system that target cancer cells.

    The study’s main goals were to:

    • find out if this treatment is safe and kills tumor cells
    • learn what kind of side effects occur
    • study how the body uses the hu14.18K322A antibody
    • find out whether the body makes antibodies against the  hu14.18K322A

    When was this study done?

    It took place between April 2012 and August 2014.

    What did the study consist of?

    1. Immunotherapy with an antibody called hu14.18K322A
    2. Immunotherapy with NK cells from a donor (usually parent or other close relative).  
    3. Three chemo regimens that are commonly used to treat neuroblastoma

    What did we learn from this study?

    • Hu14.18K322A can be safely combined with three standard chemo regimens.
    • In the past, patients had response rates of less than 50% with the chemo used in this study. Very few patients had a complete response (no evidence of tumor). But in this trial, the overall response rate was 61.5%. More than one-third of patients had a complete response or very good partial response.
    • All patients benefited, and none had cancer that got worse during the study.
    • The one-year survival rate was 77%.
    • Because this was a pilot study that treated only a few patients, we could not tell whether the NK cells helped fight the cancer.

    What are the next research steps as a result of this study?

    This successful approach of using antibody and chemotherapy will be included in a different trial for patients with newly diagnosed high-risk neuroblastoma.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    A Pilot Trial of Humanized Anti-GD2 Monoclonal Antibody (hu14.18K322A) with Chemotherapy and Natural Killer Cells in Children with Recurrent/Refractory Neuroblastoma. Federico SM, McCarville MB, Shulkin BL, Sondel PM, Hank JA, Hutson P, Meagher M, Shafer A, Ng CY, Leung W, Janssen WE, Wu J, Mao S, Brennan RC, Santana VM, Pappo AS, Furman WL. Clin Cancer Res. 2017 Nov 1;23(21):6441-6449.
    https://www.ncbi.nlm.nih.gov/pubmed/28939747

  2. Results summary of St. Jude clinical trial:

    Treatment of patients with newly diagnosed medulloblastoma, supratentorial primitive neuroectodermal tumor, or atypical teratoid rhabdoid tumor (SJMB03)

    Why was this study done?

    SJMB03 studied two different ways of giving radiation therapy, along with chemotherapy (chemo) and stem cell transplant. The goal was to find out how well they work in patients with newly diagnosed medulloblastoma, primitive neuroectodermal tumor or atypical teratoid rhabdoid tumor.

    Combining radiation therapy with chemo may kill more tumor cells. Autologous stem cell transplant may be able to replace blood-forming cells that were destroyed by these treatments. It is not yet known which radiation therapy regimen combined with chemo and transplant is more effective in treating these brain tumors.

    The main goal of this study is to learn as much as possible about the biology of medulloblastoma and related tumors, and the side effects of treatment. We will use that information to develop new and better ways to improve treatments.

    The study also sought to answer these questions:

    • Can a new computer-based educational program help prevent at least some of the loss in reading, language and learning skills?
    • Will changes in MR images of the brain help identify patients who are more likely to have problems with learning and functioning?
    • Will a lower dose of radiation to the brain still cure the tumor?
    • What are the side effects of the treatment?
    • How can MR imaging help us learn more about how to diagnose, treat and monitor these diseases?
    • How does the body break down and remove certain drugs?
    • What can we learn about this treatment by studying the spinal fluid of patients?

    When was this study done?

    The study opened in August 2003 and closed to accrual in March 2013.

    What did the study consist of?

    1. Radiation treatments to the brain and spine
    2. Collection of blood stem cells for later use with chemo
    3. Treatment with high-dose chemo (cisplatin, vincristine and cyclophosphamide) and stem cell transplant
    4. Education

    What did we learn from this study?

    One of the important things we learned in this study was about the risk of hearing loss to children who receive the chemo drug cisplatin.

    Scientists thought differences in patients’ genetic makeup might affect the chances of having hearing loss. The scientists checked the DNA (genetic make-up) of patients in this study and an earlier study. The researchers looked for more than 1.7 million common inherited variations in the genes.

    The search led to a gene called ACYP2. Some versions of this gene were linked with more than a four-fold greater risk of hearing loss. The findings may help us find out patients who are more likely to develop hearing loss due to cisplatin treatment.

    What are the next research steps as a result of this study?

    Only 1 in 8 patients with cisplatin-related hearing loss carried the ACYP2 gene variant. This suggests that other genes that we have not discovered yet also contribute to hearing loss. More research is needed to understand how the ACYP2 variations affect hearing loss from cisplatin.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    Common variants in ACYP2 influence susceptibility to cisplatin-induced hearing loss. Xu H, Robinson GW, Huang J, Lim JY, Zhang H, Bass JK, Broniscer A, Chintagumpala M, Bartels U, Gururangan S, Hassall T, Fisher M, Cohn R, Yamashita T, Teitz T, Zuo J, Onar-Thomas A, Gajjar A, Stewart CF, Yang JJ.. Nat Genet 2015;47:263-6.
    https://www.ncbi.nlm.nih.gov/pubmed/25665007

  3. Results summary of St. Jude clinical trial:

    Risk-adapted therapy for young children with medulloblastoma (SJYC07)

    Why was this study done?

    SJYC07 used a new treatment for brain tumors in children under age 5 years. Doctors used a new combination of standard chemotherapy (chemo) drugs. Some children also had radiation therapy.

    Radiation therapy is usually given to the brain and spine of children with medulloblastoma. Very young children who get this treatment may have lasting problems with thinking, learning and growing. In this study we delayed or avoided using radiation therapy, when possible.

    Chemotherapy (chemo) is used to try to shrink medulloblastoma in young children. This allows the brain more time to develop before radiation is given. Chemo may also prevent radiation from being needed at all.

    The study’s main goals were to:

    • study how well this treatment works on brain tumors in young children
    • better identify groups of tumor types
    • help predict which patients may respond to treatment, and which patients may have a higher risk for relapse (tumor coming back)
    • learn more about the genetic makeup of brain tumors in young children  

    When was this study done?

    The study opened in November 2007 and closed in April 2017.

    What did the study consist of?

    Children in this study were treated according to their risk group: low risk, intermediate-risk or high risk.

    First, surgeons removed as much tumor as possible.

    Then all patients had four courses of induction chemo: methotrexate, vincristine, cisplatin and cyclophosphamide. Patients with high-risk disease also received vinblastine.

    Next came consolidation therapy based on risk groups:

    • Low-risk patients had two, four-week cycles of cyclophosphamide, etoposide and carboplatin.
    • Intermediate-risk patients had focal radiation therapy to the tumor bed.
    • High-risk patients had chemo with topotecan and cyclophosphamide.

    Finally, all patients received cyclophosphamide, topotecan and erlotinib.

    What did we learn from this study?

    • One out of every three children on this study was alive five years later without evidence of tumor.
    • For children in the low-risk group, the survival rate was 55%; for intermediate-risk, 24.6%; and for high-risk, 16.7%.
    • The risk-adapted approach to treatment did not improve event-free survival for all young children with medulloblastoma.
    • Studying the genetic makeup of these tumors is very important.
    • Some patients with sonic hedgehog (SHH) subgroup tumors had a unique feature in the biochemical markings (called methylation pattern) of their tumors. Those children had improved survival of 51% compared with patients whose tumors did not have the special feature.
    • We found two new subtypes within the SHH subgroup. These were named iSHH-I and iSHH-II. Children with iSHH-II had better event-free survival rates than those with iSHH-I. This outcome occurred without radiation, intraventricular chemo or high-dose chemo.

    What are the next research steps as a result of this study?

    By studying the genetic makeup of the tumors, we advanced our understanding of medulloblastoma. We will use results of SJYC07 to plan future medulloblastoma trials. What we learned will lead to new ways to classify these tumors in the future, and for better ways for us to plan new risk-adapted studies to improve the treatment for children with this disease.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial. Robinson GW, Rudneva VA, Buchhalter I, Billups CA, Waszak SM, Smith KS, Bowers DC, Bendel A, Fisher PG, Partap S, Crawford JR, Hassall T, Indelicato DJ, Boop F, Klimo P, Sabin ND, Patay Z, Merchant TE, Stewart CF, Orr BA, Korbel JO, Jones DTW, Sharma T, Lichter P, Kool M, Korshunov A, Pfister SM, Gilbertson RJ, Sanders RP, Onar-Thomas A, Ellison DW, Gajjar A, Northcott PA. Lancet Oncol. 2018 Jun;19(6):768-784. doi: 10.1016/S1470-2045(18)30204-3. Epub 2018 May 16.
    https://www.ncbi.nlm.nih.gov/pubmed/29778738

Immunodeficiency Disease Clinical Trials

  1. Results summary of St. Jude clinical trial:

    A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells (LVXSCID-ND)

    Why was this study done?

    X-linked severe combined immunodeficiency (X-SCID) is a disorder that is inherited (passed down through families). X-SCID is caused by a change (mutation) in a gene that is involved in the immune system. Children with X-SCID have problems fighting off infections.

    Past treatments used bone marrow transplants from matched-sibling (brother or sister) donors. But 80% of patients do not have a perfect match. This study is designed to help infants with X-SCID who lack matched donors.

    The study evaluates a treatment called lentiviral gene transfer. This involves taking bone marrow stem cells from the child and transferring a normal (corrected) copy of the gene into these cells in the lab. The child then receives a low dose of a chemotherapy (chemo) drug called busulfan, to make room in the bone marrow, before the stem cells with the corrected gene copy are given back.

    The study’s main goals were to find out if:

    • lentiviral gene transfer is safe
    • it can be done using the methods St. Jude developed
    • it can provide a normal immune system for the patient
    • there are long-term effects of the treatment
    • it is safe and effective to use busulfan as part of the therapy

    When was this study done?

    The study opened in August 2016 and is scheduled to close in May 2022.

    What did the study consist of?

    • Tests were done to find out if this study was a good option for the child.
    • Clinicians collected bone marrow cells from the child. These cells contained the mutated gene.
    • A corrected version of the gene was inserted into the collected cells.
    • The child received busulfan to make room for the new cells.
    • The cells with the inserted gene were given back into the child’s vein.
    • The medical team watched the child closely until the immune system recovered.
    • Ten-year follow-up began after the child left the hospital.

    What did we learn from this study?

    The first eight infants in this study are producing functional immune cells for the first time.

    Early results show that low doses of busulfan increase engraftment of gene-corrected stem cells in the bone marrow without causing the side effects found with standard doses.

    All eight patients who had infections before gene therapy have recovered. They are developing and growing normally. None has developed a life-threatening infection since receiving gene therapy. No patients have developed leukemia, a side effect of an earlier gene therapy study done elsewhere for X-SCID.

    What are the next research steps as a result of this study?

    This study will continue to enroll infants, so we can learn more about lentiviral gene therapy and low-dose busulfan use.

    How does this study affect my child?

    Every survivor of X-SCID gene therapy should receive long-term follow-up care. Your child will receive information and guidance for care. Please speak with your doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. E. Mamcarz, S. Zhou, T. Lockey, H. Abdelsamed, S.J. Cross, G. Kang, Z. Ma, J. Condori, J. Dowdy, B. Triplett, C. Li, G. Maron, J.C. Aldave Becerra, J.A. Church, E. Dokmeci, J.T. Love, A.C. da Matta Ain, H. van der Watt, X. Tang, W. Janssen, B.Y. Ryu, S.S. De Ravin, M.J. Weiss, B. Youngblood, J.R. Long‑Boyle, S. Gottschalk, M.M. Meagher, H.L. Malech, J.M. Puck, M.J. Cowan, B.P. Sorrentino. N Engl J Med. 2019 Apr 18; 380(16): 1525–1534. 
    https://www.ncbi.nlm.nih.gov/pubmed/30995372

Infectious Diseases Clinical Trials

  1. Results summary of St. Jude clinical trial: 

    Catheter Resistance Monitoring to Predict Catheter-Associated Adverse Events in Children and Adolescents (CARMA)

    Why was this study done?

    Central venous lines (catheters) make the treatment of cancer much easier, but blockages can occur in these lines. Children with blockages sometimes need to have their central lines replaced. These patients may also have a higher chance of getting blood infections or large blood clots in their veins.

    The study’s main goals were to:

    • Find out whether blockages in the central line could be predicted by measuring the pressure in the line at different flow rates
    • Learn how families feel about the process

    When was this study done?

    The study opened in December 2012 and closed in September 2016.

    What did the study consist of?

    • Clinicians measured pressure in the central venous line once a week for 12 weeks
    • Staff flushed the line and then gave a small amount of fluid using a standard IV pump. During this time, clinicians measured pressure in the line several times 
    • Researchers also collected information about the child’s health before, during and for up to three weeks after
    • Four times during the study, families took a survey to find out how they felt about the procedure and its impact on their lifestyles

    What did we learn from this study?

    The main result of the study was that some episodes of catheter blockage were predictable up to 10 days before they occurred, but others were not. The good news is that test performed much better than chance, so is still promising. Overall, families thought the measurement process was acceptable. 

    What are the next research steps as a result of this study?

    Scientists could work to improve the resistance monitoring technique and test it again in a new group of patients. In the long run, we hope to improve treatment of all children with cancer by preventing central line problems.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Monitoring Central Venous Catheter Resistance to Predict Imminent Occlusion: A Prospective Pilot Study. Wolf J, Tang L, Rubnitz JE, Brennan RC, Shook DR, Stokes DC, Monagle P, Curtis N, Worth LJ, Allison K, Sun Y, Flynn PM. PLoS One. 2015 Aug 31;10(8):e0135904.
    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4555832/

  2. Results summary of St. Jude clinical trial:

    Ethanol Lock Therapy for Treatment and Secondary Prophylaxis of Central Line-Associated Bloodstream Infection (ETHEL)

    Why was this study done?

    Central venous lines and ports make cancer treatment much easier, but they increase the chances of getting blood infections. A line infection occurs when bacteria or fungus attach to the line. Children with these infections generally have to stay in the hospital and receive antibiotics or antifungals to help clear the infection. But, sometimes it is not possible to clear the infection, or the infection returns after treatment. Once a child has a line infection, the chance of getting another one is much higher.

    We wanted to find out if we could improve the chance of curing current line infections and preventing others by using a treatment called ethanol lock therapy or ELT. Ethanol works to kill germs that might cause an infection. ELT involves injecting a solution of ethanol and water into the line or port. After a dwell period, the solution is removed.

    The study’s main goals were to:

    • See if giving ethanol locks could improve central line infection outcomes by reducing the number of people who had their lines removed or had recurrence of the infection.

    When was this study done?

    The study opened in December 2011 and closed in November 2016.

    What did the study consist of?

    Patients took part in the study for about nine months, including five days in the “treatment” part, six months in the “prevention” part and up to 90 days in the “follow-up” part. 

    Treatment included:

    • Ethanol or heparin-saline lock-therapy each day
    • A blood culture every day for 5 days to check for bacteria in the blood
    • A test to measure the level of ethanol in the blood   

    Prevention included:

    • For patients with Hickman or Broviac lines: lock-therapy three times a week
    • For patients with ports: lock-therapy only when ports were accessed
    • For all patients: blood tests to monitor for side effects about once per month

    Follow-up included:

    • Monitoring for 90 days to find out if any line infections occurred after lock-therapy stopped

    Patients in this study were assigned randomly to receive either ELT or a heparin-saline therapy. The study was ”blinded,” meaning that neither patients nor the staff running the study knew who received which therapy until the study was over.

    What did we learn from this study?

    Ethanol lock therapy did not prevent infections or keep infections from coming back. In fact, the risk of treatment-failure was the same in each group. While there were no serious adverse events related to the locks, the therapy increased the number of blockages in the lines.  

    What are the next research steps as a result of this study?

    Even though these results were surprising, the study answered an important research question that will help us treat these infections by reducing the use of an unhelpful treatment. Researchers continue to explore the best ways to prevent central-line infections.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    Treatment and secondary prophylaxis with ethanol lock therapy for central line-associated bloodstream infection in paediatric cancer: a randomised, double-blind, controlled trial. Wolf J, Connell TG, Allison KJ, Tang L, Richardson J, Branum K, Borello E, Rubnitz JE, Gaur AH, Hakim H, Su Y, Federico SM, Mechinaud F, Hayden RT, Monagle P, Worth LJ, Curtis N, Flynn PM. Lancet Infect Dis. 2018 Aug;18(8):854-863.
    https://www.ncbi.nlm.nih.gov/pubmed/29884572

Leukemia Clinical Trials

  1. Results summary of St. Jude clinical trial:

    Clofarabine Can Replace Anthracyclines and Etoposide in Remission Induction Therapy for Childhood Acute Myeloid Leukemia: The AML08 Multicenter, Randomized Phase III Trial

    Why was this study done?

    Nearly 500 children are found to have acute myeloid leukemia (AML) each year in the U.S. About half of these children are cured with standard therapy, which means they show no signs of leukemia for five years.

    This study’s main goal was to improve the cure rate of children and adolescents with AML.

    The study’s other goals were to:

    • Compare the response rates after one course of chemotherapy (chemo) in patients treated with the standard drug combination of cytarabine, daunorubicin and etoposide versus the experimental combination of clofarabine and cytarabine
    • Find out if natural killer (NK) cell therapy will be useful for patients with standard-risk AML
    • Learn more about the biology and genetic makeup of AML by studying blood and bone marrow samples
    • Learn more about how AML treatment drugs work in the body of patients with AML
    • Learn how the drugs affect the body and how patients’ own genetic makeup may predict who will have more side effects and who will respond to treatment
    • Find out if we can prevent major infections by giving antibiotics and antifungal drugs when blood counts are very low

    When was this study done?

    The study opened in August 2008 and closed in March 2017.

    What did the study consist of?

    • All patients were randomly assigned to receive clofarabine and cytarabine or cytarabine, daunorubicin, and etoposide as their first treatment course
    • Low-risk patients received four courses of chemotherapy
    • Standard-risk patients who had an appropriate NK cell donor received four courses of chemotherapy followed by an infusion of NK cells. Other standard-risk patients received four courses of chemotherapy without NK cell therapy.
    • High-risk patients received two or three courses of chemotherapy followed by hematopoietic cell transplantation.

    What did we learn from this study?

    After two courses of therapy, 92.3% of the 285 patients were in complete remission. Minimal residual disease was present on Day 22 of therapy in 47% of patients who received clofarabine and cytarabine and in 35% of patients who received cytarabine, daunorubicin and etoposide. Despite this result, the 3-year event-free survival rate did not differ significantly between the two treatment arms. The 3-year overall survival rates was 75% for patients who received clofarabine and cytarabine and 65% for those who received cytarabine, daunorubicin and etoposide (result is not statistically different).

    What are the next research steps as a result of this study?

    The findings suggest that the use of clofarabine with cytarabine during remission induction might reduce the need for anthracycline and etoposide in pediatric patients with AML and may reduce rates of cardiomyopathy and treatment-related cancer.

    How does this study affect my child?

    Every survivor of AML should receive long-term follow-up care. Your child will receive information and guidance for care. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Clofarabine Can Replace Antracyclines and Etoposide in Remission Induction Therapy for Childhood Acute Myeloid Leukemia: The AML08 Multicenter, Randomized Trial. Rubnitz JE, Lacayo NJ, Inaba H, Heym K, Ribeiro RC, Taub J, McNeer J, Degar B, Schiff D, Yeoh AE, Coustan-Smith E, Wang L, Triplett B, Raimondi SC, Klco J, Choi J, Pounds S, Pui CH. J Clin Oncol. 2019 Aug 10;37(23):2072-2081.
    https://www.ncbi.nlm.nih.gov/pubmed/31246522

  2. Results summary of St. Jude clinical trial:

    Haploidentical Hematopoietic Stem Cell Transplantation Using A Novel Clofarabine Containing Conditioning Regimen For Patients With Refractory Hematologic Malignancies

    Why was this study done?

    When blood cancer relapses or does not respond to standard treatment, the cancer is difficult to treat or cure. One option for treatment is  a stem cell transplant. A family member who is not a perfect match is often used for these patients, since only one out of every three patients has a matched donor available. The transplants can occur more rapidly, since the time to find unrelated donors may take up three or four months.

    For these patients, we need new treatments that kill the leukemia cells with fewer long-term side effects and quicker immune recovery time.

    We wanted to study the safety of a chemo drug called clofarabine when given with a stem cell transplant. We also wanted to find out the highest dose of clofarabine that can be given safely with a stem cell transplant and whether the transplant would work.

    The study’s main goals were to:

    • improve the survival rate
    • find out the safest dose of clofarabine that can be given
    • learn how the donor stem cells begin to grow in the body after transplant
    • find out how often children who receive this therapy get graft-versus-host disease (GVHD)
    • find out the effects (good and bad) of this procedure
    • see if there is a difference in the immune system of patients who develop GVHD as compared to those who do not develop GVHD

    When was this study done?

    The study opened in January 2009 and closed in October 2016.

    What did the study consist of?

    • The patient received a conditioning treatment that included clofarabine.
    • A family member donated stem cells.
    • The stem cells were processed and filtered.
    • These stem cells were transplanted (infused) into the patient.

    What did we learn from this study?

    Patients tolerated clofarabine well at a dose of 50 mg/m2  given through the vein every day for five days. All patients had rapid recovery of their blood cells after the stem cells from the donor populated their bone marow. 

    Only two of the 13 patients in the study died of treatment-related causes. Unfortunately, the relapse rate (recurrence of the disease) remained very high.

    What are the next research steps as a result of this study?

    Higher doses of clofarabine seemed to be more effective at controlling disease than lower doses. A study with a larger group of patients is needed to further explore that possibility.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    Haploidentical Donor Transplantation Using a Novel Clofarabine-Containing Conditioning Regimen for Very High-Risk Hematologic Malignant Neoplasms. Sharma A, Kang G, Sunkara A, Hiroto Inaba H, Jeha S, Cross SJ, Geiger T, Triplett B. J Pediatr Hematol Oncol. 2018 Nov; 40(8): e479–e485.
    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6197927

  3. Results summary of St. Jude clinical trial:

    Testing the addition of a new drug, selinexor (KPT-330), to chemotherapy in patients with advanced leukemia or myelodysplastic syndrome

    Why was this study done?

    Cancer is the uncontrolled growth of human cells. The growth of normal human cells is controlled by multiple processes. When cancer cells escape one or more of these processes, it leads to rapid growth.

    One way cancer cells continue to grow is by getting rid of certain proteins called “tumor suppressor proteins” that normally cause cancer cells to die. Selinexor (KPT-330) works by trapping these proteins within the cell. That causes the cancer cells to die or stop growing.

    This study was done to test the safety of selinexor and to find the highest dose of the drug that can be given safely when it is combined with two other chemotherapy (chemo) drugs. The study tested different doses of selinexor to see which dose is safer in children with advanced leukemia or myelodysplastic syndrome (MDS).

    The study was done in two parts: Phase I and Phase II. The Phase I study aimed to find the highest safe dose of selinexor that could be given when combined with the other two drugs. The Phase II portion looked at how active was the drug combination in making the leukemia go away in patients with leukemia or MDS.

    The study’s main goals were to find:

    • the highest safe dose of selinexor that could be given when combined with fludarabine and cytarabine
    • what side effects resulted when the drug combination was given
    • how the body absorbs, processes and removes the drug
    • how treatment with the drug affects the body

    When was this study done?

    The study opened August 2014 and closed in July 2019.

    What did the study consist of?

    • Patients took the drug selinexor by mouth along with the intravenous drugs fludarabine and cytarabine.
    • Different doses of selinexor were given to groups of patients.
    • The doses increased for each group in the study until side effects required the dose to be lowered. This final dose was used in Phase II.

    What did we learn from this study?

    The study found that six doses of selinexor, given at 55 milligrams/m2 per dose, was well tolerated in combination with the two chemo drugs in children with acute leukemia that had come back or was hard to treat. The most common side effect was a lowering of sodium in the blood (not causing any symptoms). Two patients treated at higher doses also had transient side effects of difficulty talking, walking or coordination of movement.

    In almost half (47%) of the patients treated we also observed that the leukemia cells responded to the treatment. This study and other promising studies show the combo is effective in high-risk leukemia and leukemia cells. Scientists will conduct more research to test the usefulness of selinexor-based therapy in children.

    What are the next research steps as a result of this study?

    The Phase II portion of this study further explored the combination of selinexor, fludarabine and cytarabine and its effects.

    How does this study affect my child?

    Although survival rates are near 90% for children with acute lymphoblastic leukemia and 60% for those with acute myeloid leukemia, patients with relapsed or hard-to-treat disease have dismal outcomes. Response rates to selinexor are promising and will be further studied.

    Your child will receive information and guidance for care. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    Phase I Study of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination With Fludarabine and Cytarabine, in Pediatric Relapsed or Refractory Acute Leukemia. Thomas B. Alexander, John K. Choi, Raul C. Ribeiro, Ching-Hon Pui, and Jeffrey E. Rubnitz, St Jude Children's Research Hospital; Raul C. Ribeiro, Ching-Hon Pui, and Jeffrey E. Rubnitz, University of Tennessee College of Medicine, Memphis, TN; and Norman J. Lacayo, Lucile Packard Children's Hospital Stanford, Stanford, CA. J Clin Oncol. 2016 Dec;34(34):4094-4101.
    https://www.ncbi.nlm.nih.gov/pubmed/27507877

  4. Results summary of St. Jude clinical trial:

    TOTAL Therapy Study XV for Newly Diagnosed Patients with Acute Lymphoblastic Leukemia (TOTXV)

    Why was this study done?

    TOTXV is the 15th in a series of studies for children with acute lymphoblastic leukemia (ALL). The TOTAL studies began in 1962 at St. Jude. Each study has built on results from past Total studies, new discoveries by our clinical investigators and laboratory scientists, and advances from other groups treating childhood leukemia around the world.

    Earlier studies showed that most children with ALL can have a long-term remission (no evidence of disease) if they get optimal doses and combinations of several chemotherapy (chemo) drugs. Some treatments in the past also included radiation to the brain.

    The study’s main goals were to:

    • pioneer the use of response to the first six weeks of chemo as determined by minimal residual disease. Minimal residual disease is a test to measure how much leukemia is left in the body. We used that information to adjust the chemo and dosages that came after that point. This is the way that we can identify patients who need more intense treatment for cure and those who can be successfully treated with less chemo to reduce acute and long-term side effects.   
    • pioneer the use of chemo given into the cerebrospinal fluid and blood to replace radiation to brain. We did this to prevent and treat leukemia in the central nervous system.
    • learn if the amount of the drug methotrexate that gets into leukemia cells is related to the child’s leukemia subtype, genetic make-up, or length of methotrexate infusion

    We also studied the side effects of treatment.

    When was this study done?

    The study opened in June 2000 and closed to accrual in August 2008.

    What did the study consist of?

    • Children first received four days of methotrexate to find out if the length of infusion affects how well the drug works. 
    • Next came remission induction therapy. Several strong drugs were used to kill leukemia cells in the bone marrow and to allow normal blood cells to begin to grow again. The goal was to have no evidence of leukemia detectable in blood or bone marrow (remission).
    • For the next phases of treatment, patients were treated according to their risk group: low risk, standard risk or high risk.
    • Children in TOTXV received many drugs, including glucocorticoids (commonly known as ‘steroids’) during remission induction and maintenance therapy. Glucocorticoids are a crucial part of therapy. They are man-made versions of steroids that occur naturally in your body. Some of the glucocorticoids used in TOTXV included prednisone, dexamethasone and hydrocortisone.

    What did we learn from this study?

    One side effect of glucocorticoids is osteonecrosis. In this condition, joint bone tissue dies from lack of blood supply. This is one of the more common therapy-related side effects of ALL treatment.

    St. Jude scientists wanted to know if certain children in TOTXV had genetic variations passed down through their families that increased their risk of osteonecrosis. By studying the complete DNA sets, or genomes, of patients, scientists found that:

    • patients of European ancestry, especially girls, were at higher risk of having osteonecrosis
    • it happens most often in children over 10 years old
    • it usually occurs within the first two years of treatment
    • certain inherited variations are involved in the development of this condition

    What are the next research steps as a result of this study?

    Because of this study, scientists now know where the variations are located and the genes that are involved. The researchers are studying in laboratory models of osteonecrosis how these genes contribute to this complication of treatment. Through these studies, scientists hope to develop ways to decrease osteonecrosis in children who have these genetic variations.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Genetics of glucocorticoid-associated osteonecrosis in children with acute lymphoblastic leukemia. Karol SE, Yang W, Van Driest SL, Chang TY, Kaste S, Bowton E, Basford M, Bastarache L, Roden DM, Denny JC, Larsen E, Winick N, Carroll WL, Cheng C, Pei D, Fernandez CA, Liu C, Smith C, Loh ML, Raetz EA, Hunger SP, Scheet P, Jeha S, Pui CH, Evans WE, Devidas M, Mattano LA Jr, Relling MV.Blood. 2015 Oct 8;126(15):1770-6.
    https://www.ncbi.nlm.nih.gov/pubmed/26265699

    Treating childhood acute lymphoblastic leukemia without cranial irradiation. Pui CH, Campana D, Pei D, Bowman WP, Sandlund JT, Kaste SC, Ribeiro RC, Rubnitz JE, Raimondi SC, Onciu M, Coustan-Smith E, Kun LE, Jeha S, Cheng C, Howard SC, Simmons V, Bayles A, Metzger ML, Boyett JM, Leung W, Handgretinger R, Downing JR, Evans WE, Relling MV. New England Journal of Medicine. 2009 Jun 25;360(26):2730-41.
    https://www.ncbi.nlm.nih.gov/pubmed/19553647

    Pharmacokinetic, pharmacodynamic, and pharmacogenetic determinants of osteonecrosis in children with acute lymphoblastic leukemia. Kawedia JD, Kaste SC, Pei D, Panetta JC, Cai X, Cheng C, Neale G, Howard SC, Evans WE, Pui CH, Relling MV. Blood. 2011 Feb 24;117(8):2340-7. 
    https://www.ncbi.nlm.nih.gov/pubmed/21148812

  5. Results summary of St. Jude clinical trial:

    TOTAL Therapy Study XVI for Newly Diagnosed Patients with Acute Lymphoblastic Leukemia (TOTXVI)

    Why was this study done?

    TOTXVI is the 16th in a series of studies for children with acute lymphoblastic leukemia (ALL). The TOTAL studies began in 1962 at St. Jude. Each study has built on what has been learned from past TOTAL studies, new discoveries at St. Jude, and advances from other groups treating childhood leukemia around the world.

    Earlier studies showed that most children with ALL can have a long-term remission (no evidence of disease) if they get strong chemotherapy (chemo) with several drugs. Some treatments in the past also included radiation to the brain.

    The study’s main goals were to:

    • improve the survival rates of children with acute lymphoblastic leukemia.
    • find out if a higher dose of a drug called PEG-asparaginase (PEG-ASP) would yield better results than a standard dose without more side effects
    • learn if giving more intense treatment to high-risk patients increases survival rates over past studies
    • find out if giving more intensive chemo into the fluid around the brain and spinal cord (CSF) for high-risk patients would prevent the leukemia from coming back (relapse) in the CSF
    • learn more about how the drugs are processed, eliminated and affect the body
    • test new methods to measure minimal residual disease (MRD) to help adjust the intensity of treatment to the risk of relapse
    • use stool samples to learn more about preventing and treating infections in children and young adults with ALL
    • learn more about how drugs affect the brain’s structure and function, and how they are influenced by genetic factors

    When was this study done?

    The study opened in October 2007 and closed to accrual in March 2017.

    What did the study consist of?

    There are three main stages of treatment (remission induction, consolidation and continuation):

    • Remission induction therapy uses several strong drugs to kill leukemia cells in the bone marrow and to allow normal blood cells to begin to grow again. The goal is remission, in which there is no evidence of leukemia.
    • The next stage, called consolidation, tries to deliver a “knock-out punch” to any remaining leukemia cells.
    • In the final stage, called maintenance, we try to keep the leukemia from coming back.
    • Patients received different treatments according to their risk group: low risk, standard risk or high risk.

    What did we learn from this study?

    • We learned that excellent control of leukemia in the brain can be achieved without using cranial irradiation. That finding applies even to patients with high-risk subtypes. Optimal chemo, including three drugs given in the cerebrospinal fluid, can prevent relapses in the brain without excessive toxicity.
    • We learned the importance of giving antibiotics to prevent infections. Infection is one of the most common causes of death in children being treatment for ALL. Most life-threatening infections occur during the induction phase. Preventive antibiotic treatment has been controversial because of worries about whether it’s needed or if it may cause antibiotic resistance. In the largest study of its kind, some patients in TOTXVI received no preventive antibiotics during induction. A second group got an antibiotic called levofloxacin. A third group got other antibiotics. Scientists learned that preventive antibiotics reduced the chance of serious infections by more than 70%. Levofloxacin reduced the chance of Clostridium difficile infections by more than 95%. Many children develop allergies to PEG-ASP, an important drug used to treat leukemia. When that happens, they need to switch to another drug that must be given more often. Scientists wanted to find out what puts children at risk for PEG-ASP allergies. The scientists found antibodies that predict reactions to PEG-ASP. They also learned that children who had more intrathecal therapy (when the drug is infused into the fluid-filled area in the spinal cord) had fewer reactions to PEG-ASP. PEG-ASP resulted in fewer allergies than L-ASP, a similar drug that had been used in the previous TOTXV clinical trial.
    • We learned how to predict infection risk through stool samples. Scientists know that the microbes living in our gut can affect our overall health. If chemo gets these microbes out of balance, life-threatening infections can occur. Researchers collected stool samples from patients at diagnosis and after each phase of chemo. They used those samples to study the role of the microbes in the gut. The scientists’ aim was to find characteristics that predict such problems as diarrhea, bloodstream infections, fever or low numbers of white blood cells. The scientists discovered that children who have a lot of proteobacteria (a certain type of bacteria) in the gut before chemo are at a high risk of getting fever and low numbers of white blood cells. Researchers also found that high numbers of Enterococcaceae or Streptococcaceae bacteria at any time during chemo predicts future infections and diarrhea.

    What are the next research steps as a result of this study?

    As a result of research on preventive antibiotics, scientists now suggest using levofloxacin as a preventive measure in children who undergo induction therapy for ALL. The researchers suggest close long-term monitoring of antibiotic resistance in these children.

    As a result of the research into PEG-ASP, clinicians now have a better understanding of allergic reactions to this drug, which will help predict which patients cannot take this drug.

    From the study on the gut microbiome, we learned better ways to predict which patients have a high risk of infections. Customized treatments can now be designed to lessen this risk.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Levofloxacin Prophylaxis During Induction Therapy for Pediatric Acute Lymphoblastic Leukemia. Wolf J, Tang L, Flynn PM, Pui CH, Gaur AH, Sun Y, Inaba H, Stewart T, Hayden RT, Hakim H, Jeha S. Clin Infect Dis. 2017 Nov 13;65(11):1790-1798.
    https://www.ncbi.nlm.nih.gov/pubmed/29020310

    Antibodies Predict Pegaspargase Allergic Reactions and Failure of Rechallenge. Liu Y, Smith CA, Panetta JC, Yang W, Thompson LE, Counts JP, Molinelli AR, Pei D, Kornegay NM, Crews KR, Swanson H, Cheng C, Karol SE, Evans WE, Inaba H, Pui CH, Jeha S, Relling, MV. J Clin Oncol. 2019 Aug 10;37(23):2051-2061.
    https://www.ncbi.nlm.nih.gov/pubmed/31188727

    Gut Microbiome Composition Predicts Infection Risk During Chemotherapy in Children With Acute Lymphoblastic Leukemia. Hakim H, Dallas R, Wolf J, Tang L, Schultz-Cherry S, Darling V, Johnson C, Karlsson EA, Chang TC, Jeha S, Pui CH, Sun Y, Pounds S, Hayden RT, Tuomanen E, Rosch JW. Clin Infect Dis. 2018 Aug 1;67(4):541-548.
    https://www.ncbi.nlm.nih.gov/pubmed/29518185

    Improved CNS Control of Childhood Acute Lymphoblastic Leukemia Without Cranial Irradiation: St Jude Total Therapy Study 16. Jeha S, Pei D, Choi J, Cheng C, Sandlund JT, Coustan-Smith E, Campana D, Inaba H, Rubnitz JE, Ribeiro RC, Gruber TA, Raimondi SC, Khan RB, Yang JJ, Mullighan CG, Downing JR, Evans WE, Relling MV, Pui CH. J Clin Oncol. 2019 Oct 28:JCO1901692. [Epub ahead of print]
    https://www.ncbi.nlm.nih.gov/pubmed/31657981

Long-term Effects Clinical Trials

  1. Results summary of St. Jude clinical trial:

    Risk of Psychopathology and Neurocognitive Impairment in Leukemia Survivors (NEULS)

    Why was this study done?

    We wanted to find out if survivors were having problems five or more years after treatment for acute lymphoblastic leukemia (ALL). The survivors in this study had been in the Total 15 (TOTXV) clinical trial. We looked at neurocognitive problems (the ability to concentrate, remember things, process information, learn, speak and understand). We also looked at behavioral issues (attention deficit or hyperactivity disorder) and other problems such as depression and anxiety. We also wanted to learn how caregivers were affected by the stresses of caring for a child with a life-threatening illness.

    The study’s main goals were to find out:

    • How the survivor developed after cancer and therapy
    • How cancer affects the way a family unit may function
    • The number of parents or caregivers who suffer symptoms of depression or anxiety
    • The difficulties parents or caregivers face while caring for a child with a life-threatening illness
    • How energy levels may be affected in survivors who received ALL treatment
    • How survivors’ sleep habits could be affected

    When was this study done?

    The study opened in February 2010 and closed in December 2014.

    What did the study consist of?

    • Caregivers answered questions about the stresses of caring for an ALL survivor.
    • Survivors completed neurocognitive testing and a brain MRI.

    What did we learn from this study?

    Survivors in this study were at higher risk for long-term neurocognitive and behavioral problems than the general population. The ALL survivors were more likely to have problems with memory span, processing speed and executive function (mental flexibility, fluency and planning).

    Patients who had leukoencephalopathy during treatment were at even higher risk of problems. This disorder damages the brain's white matter. White matter consists of nerve fibers covered by myelin. Damage to white matter can make it harder for different areas of the brain to work with one another. Survivors with the executive function problems also had areas of their brain that were not working efficiently. These brain areas seemed to be underdeveloped.

    What are the next research steps as a result of this study?

    We have already started another study to stimulate the underdeveloped brain areas to find out if the will work together more efficiently and if this leads to improved neurocognitive function.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment.

    Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    Leukoencephalopathy and long-term neurobehavioural, neurocognitive, and brain imaging outcomes in survivors of childhood acute lymphoblastic leukaemia treated with chemotherapy: a longitudinal analysis. Cheung YT, Sabin ND, Reddick WE, Bhojwani D, Liu W, Brinkman TM, Glass JO, Hwang SN, Srivastava D, Pui CH, Robison LL, Hudson MM, Krull KR. Lancet Haematol. 2016 Oct;3(10):e456-e466.
    https://www.ncbi.nlm.nih.gov/pubmed/27658980

Psychology Clinical Trials

  1. Results summary of St. Jude clinical trial:

    Sperm Banking Among Adolescents Newly Diagnosed with Cancer: Development of a Profiling and Referral Tool (SBANK10)

    Why was this study done?

    Survival rates among pediatric cancer patients have increased to more than 80% since the 1960s. But the life-saving therapies can lead to many chronic health conditions, including infertility. Results from the Childhood Cancer Survivor Study show that 46% of male childhood cancer survivors are infertile. As early as adolescence, cancer patients report having children among their top three life goals. They are concerned that treatment affects that possibility.

    In this study researchers wanted to learn what factors are important to cancer patients and their families when making decisions about banking sperm to preserve fertility.

    The study’s main goals were to:

    • Learn what factors best predict why adolescents newly diagnosed with cancer choose to bank sperm or decide against it
    • Use these factors to develop a tool to increase sperm banking among these patients

    When was this study done?

    The study opened in July 2010 and closed in June 2014.

    What did the study consist of?

    The study consisted of separate 20-minute surveys for the patient and their parent or caregiver.

    The patients:

    • Completed the surveys during the first week after beginning cancer treatment
    • Answered questions on their general thoughts about banking sperm
    • Answered questions about sperm banking concerns and benefits as well as their thoughts about the risk of infertility
    • Answered questions regarding their development and readiness to take part in sperm banking

    The parent or guardian completed a separate survey.

    What did we learn from this study?

    Parents’ opinions on sperm banking play an important role among adolescent males who are newly diagnosed with cancer. When a parent supports their son to bank sperm it is more likely that the adolescent will attempt sperm banking. The study also showed the value of clear communication among parents, patients and health care teams when discussing banking options. In addition, parents’ beliefs about their ability to encourage sperm banking should be considered in future studies.

    What are the next research steps as a result of this study?

    Scientists will continue to study ways to improve the health-related quality of life and fertility options for children receiving cancer treatment.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Parental influences on sperm banking attempts among adolescent males newly diagnosed with cancer. Klosky JL, Flynn JS, Lehmann V, Russell KM, Wang F, Hardin RN, Eddinger JR, Zhang H, Schenck LA, Schover LR. Fertil Steril. 2017 Dec;108(6):1043-1049. doi: 10.1016/j.fertnstert.2017.08.039.
    https://www.ncbi.nlm.nih.gov/pubmed/29202957

Quality of Life Clinical Trials

  1. Results summary of St. Jude clinical trial:

    Physical Activity to Modify Sequelae and Quality of Life in Childhood Acute Lymphoblastic Leukemia (PAQOL)

    Why was this study done?

    Children with acute lymphoblastic leukemia (ALL) are at risk for problems with their bones, muscles, nerves and heart. These problems can affect their quality of life. We wanted to find out if exercise or physical activity could prevent or improve these health problems in newly diagnosed patients.

    The study’s main goals were to:

    • Learn whether a physical activity program could improve the patients’ strength, flexibility, endurance and bone density
    • Find out whether quality of life, physical function and bone density are affected by changes in motivation and feelings about things going on in their lives
    • Look at how gender, age, family income and parent’s education affect the patients’ physical function, bone density and health-related quality of life

    When was this study done?

    The study opened in November 2009 and closed in May 2015.

    What did the study consist of?

    Each child took part in the study for about 2.5 years. Patients were randomly assigned to one of two groups. Some received a broader and more detailed exercise program. Others received a more focused exercise program.

    The patients:

    • Had scans to measure their bone strength  
    • Received home exercise programs to do about five times a week
    • Completed activity diaries
    • Wore accelerometers three times for seven days each time. These small monitors are worn on the upper arm to measure activity levels.
    • Met regularly with a physical therapist and a nurse
    • Had tests of joint flexibility, muscle strength, balance, coordination. Clinicians also timed the distance patients could walk in six minutes.   
    • Completed four surveys about their health, exercise and feelings.

    The parent or guardian also completed a questionnaire four times.

    What did we learn from this study?

    Patients must spend two to three years in therapy during important developmental periods. Children with newly diagnosed ALL had problems with bone density, body composition, strength and fitness. To improve their quality of life, the children should begin right away on a program that includes strength exercises and aerobic conditioning. This will help prevent bone loss and preserve muscle mass.

    Children with acute leukemia were also more likely to be overweight than their healthy peers. To have the most impact, these patients should begin nutrition and physical activity discussions, counseling and interventions soon after treatment begins.

    What are the next research steps as a result of this study?

    Scientists will continue to study ways to improve the health-related quality of life for children receiving cancer treatment.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Skeletal, neuromuscular and fitness impairments among children with newly diagnosed acute lymphoblastic leukemia. Ness KK, Kaste SC, Zhu L, Pui CH, Jeha S, Nathan PC, Inaba H, Wasilewski-Masker K, Shah D, Wells RJ, Karlage RE, Robison LL, Cox CL. Leuk Lymphoma. 2015 Apr;56(4):1004-11.
    https://www.ncbi.nlm.nih.gov/pubmed/25030039

Sickle Cell Disease Clinical Trials

  1. Results summary of St. Jude clinical trial:

    Long-term Effects of Erythrocyte Lysis (ELYSIS)

    Why was this study done?

    Heart disease is the main cause of death in people with sickle cell disease. We wanted to find out if chronic hemolysis (nonstop breakdown of red blood cells) causes people with sickle cell disease and other blood disorders to develop pulmonary hypertension (high blood pressure in the lungs) and other medical problems. Pulmonary hypertension is closely associated with heart failure.

    The study’s main goals were to find out:

    • If chronic hemolysis is connected to the development of pulmonary hypertension
    • The number of children or adults with hemolysis who have pulmonary hypertension
    • The spleen’s role in the development of pulmonary hypertension
    • If blood and urine test results can be related to the development of pulmonary hypertension
    • If there are genes in the blood that are important in developing pulmonary hypertension
    • If blood tests, urine tests and a heart ultrasound change over a two year period

    When was this study done?

    The study opened in March 2009 and closed in June 2016.

    What did the study consist of?

    The study involved two blood samples, two urine samples and two echocardiograms (heart ultrasounds) done two years apart.

    What did we learn from this study?

    When we looked at two key measures of heart function based on the echocardiograms, there were important changes. Measuring the elevated tricuspid valve regurgitation jet velocity (TRV) is a way to find out if there is build-up of pressure in the lungs. TRV is related to a higher risk of dying in adulthood. We found out that TRV elevation is influenced by the degree of anemia a person with sickle cell disease has. Also, measuring global longitudinal strain (GLS) is a way to find out how well the heart is working. This test is done with an echocardiogram. GLS can reveal changes in heart function for patients who have sickle cell disease. In our study, GLS of the heart’s right ventricle (RVGLS) was higher in children with sickle cell disease than in healthy individuals. Abnormal RVGLS has been linked to serious medical problems and death in patients who have heart disease. The increased RVGLS points to an early response of the right ventricle to heart problems.

    What are the next research steps as a result of this study?

    Long-term follow-up is needed to find out if GLS can help us identify heart problems early on in patients with sickle cell disease.  

    How does this study affect my child?

    Every individual with sickle cell disease should receive life-long follow-up care. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    Ventricular global longitudinal strain is altered in children with sickle cell disease. Whipple NS, Naik RJ, Kang G, Moen J, Govindaswamy SD, Fowler JA, Dowdy J, Penkert R, Joshi VM, Hankins JS. Br J Haematol. 2018 Dec;183(5):796-806.
    https://ncbi.nlm.nih.gov/pubmed/30450553

    Elevated tricuspid regurgitation velocity in congenital hemolytic anemias: Prevalence and laboratory correlates. Yates AM, Joshi VM, Aygun B, Moen J, Smeltzer MP, Govindaswamy D, Dowdy J, Cotton A, Kang G, Ware RE, Hankins JS. Pediatr Blood Cancer. 2019 Jul;66(7).
    https://www.ncbi.nlm.nih.gov/pubmed/30907497

Solid Tumor Clinical Trials

  1. Results summary of St. Jude clinical trial:

    A study of bevacizumab, a humanized monoclonal antibody against vascular endothelial growth factor (VEGF), in combination with chemotherapy for treatment of osteosarcoma (OS2008)

    Why was this study done?

    Children and adolescents with osteosarcoma, a type of bone cancer, usually undergo surgery and take multiple cancer drugs: methotrexate, doxorubicin (brand name: Adriamycin®) and cisplatin. This standard chemotherapy (chemo) for osteosarcoma is called MAP, formed from letters in the names of the drugs.

    Osteosarcoma is the most common cancerous bone tumor in children and teens. The outcome for patients whose disease has not spread has not improved for decades. New therapies are needed.

    One strategy targets a substance made by cells called vascular endothelial growth factor (VEGF). VEGF plays a key role in a process called angiogenesis. Angiogenesis is the formation of new blood vessels. This process spurs tumor growth and helps cancer cells spread to other parts of the body. Survival rates are poor for osteosarcoma patients with high levels of VEGF.

    The drug bevacizumab can bind to cancer cells and prevent blood vessel formation and tumor growth.

    Scientists wanted to know if they could improve survival by combining bevacizumab and MAP to stop angiogenesis.

    The study’s main goals were to:

    • find out if the anti-VEGF drug bevacizumab could enhance MAP chemo
    • find out if bevacizumab affects healing after surgery. The natural formation of new blood vessels is important in these patients after surgery

    When was this study done?

    The study opened in June 2008 and closed to accrual May 2012.

    What did the study consist of?

    Thirty-one patients received two treatment courses of MAP chemo before and after surgery. They got bevacizumab before starting chemo and at specific times beginning five weeks after surgery. Also, scientists studied how the amount of bevacizumab changed with time within the body.

    Clinicians monitored patients for bevacizumab-related side events. In other adult studies side effects included high blood pressure, blood clots, blocked arteries, and higher-than normal amounts of protein in the urine.

    What did we learn from this study?

    • Bevacizumab did not enhance standard chemo and improve patient outcomes.
    • Adding bevacizumab to standard chemo did not add significant side effects to treatment. Most side effects from bevacizumab were relatively mild.
    • Bevacizumab increased the risk of wound-healing problems. Problems occurred in about half of the patients after surgery. Twenty-one percent of planned doses of the drug were stopped or skipped.

    What are the next research steps as a result of this study?

    Bevacizumab will not be combined with MAP in the future for children and adolescents with osteosarcoma. The study provides important data that can be used for future studies of antiangiogenic drugs in treatments that require surgery for bone tumors.

    Results of this clinical trial will help clinicians as they evaluate other newer antiangiogenic drugs for future osteosarcoma studies.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s doctor about questions or concerns you have as a result of this study.

    Publications generated from this study:

    A phase II trial evaluating the feasibility of adding bevacizumab to standard osteosarcoma therapy. Navid F, Santana VM, Neel M, McCarville MB, Shulkin BL, Wu J, Billups CA, Mao S, Daryani VM, Stewart CF, Kunkel M, Smith W, Ward D, Pappo AS, Bahrami A, Loeb DM, Reikes Willert J, Rao BN, Daw NC. Int J Cancer. 2017 Oct 1;141(7):1469-1477. 
    https://www.ncbi.nlm.nih.gov/pubmed/28631382

    Population pharmacokinetics of bevacizumab in children with osteosarcoma: implications for dosing. Turner DC, Navid F, Daw NC, Mao S, Wu J, Santana VM, Neel M, Rao B, Willert JR, Loeb DM, Harstead KE, Throm SL, Freeman BB 3rd, Stewart CF. Clin Cancer Res. 2014 May 15;20(10):2783-92. 
    https://www.ncbi.nlm.nih.gov/pubmed/24637635

  2. Results summary of St. Jude clinical trial:

    A safety/feasibility trial of the addition of the humanized anti-GD2 antibody (HU14.18K322A) with and without natural killer cells to chemotherapy in children and adolescents with recurrent/refractory neuroblastoma (GD2NK)

    Why was this study done?

    GD2NK used an antibody called hu14.18K322A and natural killer (NK) cells in patients who have neuroblastoma that has come back or is hard to treat. This treatment was given along with chemotherapy (chemo).

    Our immune system looks for and attacks cells that it sees as harmful, such as bacteria, viruses and cancer cells. Antibodies are part of the immune system. Hu14.18K322A is a monoclonal antibody, a protein made in a lab. We designed hu14.18K322A to bind to cancer cells that contain or “express” a molecule called an anti-GD2 antigen. Almost all neuroblastoma cells express this antigen. When hu14.18K322A binds to these types of cancer cells, it tells the immune system to attack and kill the cancer cells without harming nearby healthy cells.

    NK cells are also special cells in our immune system that target cancer cells.

    The study’s main goals were to:

    • find out if this treatment is safe and kills tumor cells
    • learn what kind of side effects occur
    • study how the body uses the hu14.18K322A antibody
    • find out whether the body makes antibodies against the  hu14.18K322A

    When was this study done?

    It took place between April 2012 and August 2014.

    What did the study consist of?

    1. Immunotherapy with an antibody called hu14.18K322A
    2. Immunotherapy with NK cells from a donor (usually parent or other close relative).  
    3. Three chemo regimens that are commonly used to treat neuroblastoma

    What did we learn from this study?

    • Hu14.18K322A can be safely combined with three standard chemo regimens.
    • In the past, patients had response rates of less than 50% with the chemo used in this study. Very few patients had a complete response (no evidence of tumor). But in this trial, the overall response rate was 61.5%. More than one-third of patients had a complete response or very good partial response.
    • All patients benefited, and none had cancer that got worse during the study.
    • The one-year survival rate was 77%.
    • Because this was a pilot study that treated only a few patients, we could not tell whether the NK cells helped fight the cancer.

    What are the next research steps as a result of this study?

    This successful approach of using antibody and chemotherapy will be included in a different trial for patients with newly diagnosed high-risk neuroblastoma.

    How does this study affect my child?

    Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

    For more information

    Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

    Publication generated from this study:

    A Pilot Trial of Humanized Anti-GD2 Monoclonal Antibody (hu14.18K322A) with Chemotherapy and Natural Killer Cells in Children with Recurrent/Refractory Neuroblastoma. Federico SM, McCarville MB, Shulkin BL, Sondel PM, Hank JA, Hutson P, Meagher M, Shafer A, Ng CY, Leung W, Janssen WE, Wu J, Mao S, Brennan RC, Santana VM, Pappo AS, Furman WL. Clin Cancer Res. 2017 Nov 1;23(21):6441-6449.
    https://www.ncbi.nlm.nih.gov/pubmed/28939747

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