Clinical Trials

Diseases Treated:

Galactosialidosis

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

For patients at risk for blood clots

Eligibility:

• Participant has a diagnosis requiring anticoagulant therapy.
• Participant has completed their standard anticoagulant therapy.
• Participant is not receiving active therapy for a malignant condition.
• Participant is less than 18 years of age at the time of consent.
• Participant and legal guardian agree to food and drug restrictions during study.

Diseases Treated:

Blood clots (venous thromboembolism)

Eligibility:

This clinical trial is open only to St. Jude patients.

• Newborn to 17 years old (must be younger than 18)
• Diagnosis of blood clot in a vein (venous thromboembolism, also called VTE)
• Requires anticoagulant therapy for at least 90 days
• Received at least 5 days of heparin therapy prior to study

Diseases Treated:

Hemophilia B

Eligibility:

• Male
• At least 18 years old
• Diagnosis of hemophilia B

Diseases Treated:

Non-malignant blood diseases (non-therapeutic)

Eligibility:

• Receiving therapy or a consultation for a non-malignant blood disorder
• Biological relatives (with or without a non-malignant blood disorder) who agree to undergo genetic testing

Diseases Treated:

Sickle Cell Disease

Eligibility:

This is a non-therapeutic study for patients at St. Jude Children’s Research Hospital and Methodist Adult Comprehensive Sickle Cell Center.

• Diagnosis of sickle cell disease (SCD)
• 16 to 20 years old
• English is primary language

Diseases Treated:

X-linked Severe Combined Immunodeficiency (SCID-X1)

Eligibility:

• Diagnosis of Severe Combined Immunodeficiency, X-linked (SCID-X1)
• Newborn to 2 years of age
• No prior therapy with allogeneic stem cell transplantation
• No HIV infection

Diseases Treated:

Pyruvate kinase deficiency (PKD)

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participants of all ages with biochemically or genetically diagnosed PKD.
• Participants with a hemolytic anemia and a family member with genetically diagnosed PKD.
• The participant or the guardian of the participant is willing and able to give written informed consent and/or assent.

Diseases Treated:

Sickle cell disease

Eligibility:

This study is limited to patients living in the Memphis, Tennessee region.

Phase I

• Diagnosis of sickle cell disease
• Between 15 and 44.9 years old
• Speaks English

Phase II

• Diagnosis of sickle cell disease
• Between 15 and 44.9 years old
• Receiving hydroxyurea treatment for sickle cell disease
• Owns a smart phone
• Not currently receiving text messages for improving hydroxyurea adherence
• Not currently undergoing hydroxyurea dose escalation
• Not on any investigational new drug intervention study for sickle cell disease

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant is receiving an allogeneic hematopoietic stem cell transplant at St. Jude Children’s Research Hospital using an unlicensed cord blood unit (CBU).
• The patient has a disorder affecting the hematopoietic system that is either inherited, acquired, or a result from myeloablative treatment.

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant received an integrating retroviral or lentiviral vector based gene transfer or gene marked product at St. Jude Children’s Research Hospital within the past 15 year time period.

Diseases Treated:

• Acute lymphoblastic leukemia
• Acute myeloid leukemia
• Myelodysplasia
• Hodgkin lymphoma
• Non-Hodgkin lymphoma
• Juvenile myelomonocytic leukemia

Eligibility:

For transplant recipient:

• 21 years or younger
• Does not have a suitable sibling donor or volunteer unrelated donor
• Has a suitable single haplotype matched family member donor
• Diagnosed with high risk hematologic malignancy
• No prior allogeneic hematopoietic cell transplant

Diseases Treated:

Leukemia and other blood diseases

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant is receiving an allogeneic hematopoietic stem cell (HSC) transplant at St. Jude Children's Research Hospital using an unlicensed cord blood unit (CBU).
• Participant may be of any age and either gender.
• Participant has a medical disorder affecting the hematopoietic system that is inherited, acquired, or a result from myeloablative treatment.
• Participant is receiving HPC-CORD BLOOD product manufactured by NCBP (at least one, if the graft contains more than one units).

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

• Leukemia
• Lymphoma
• Myeloid sarcoma
• Myelodysplastic syndrome

Eligibility:

• 21 years old and younger
• Diagnosed with one of the following that has come back or did not improve after bone marrow transplant
  • Acute lymphoblastic leukemia (ALL)
  • Acute myeloid leukemia
  • Myeloid sarcoma
  • Chronic myeloid leukemia (CML)
  • Juvenile myelomonocytic leukemia (JMML)
  • Myelodysplastic syndrome (MDS)
  • Non-Hodgkin lymphoma (NHL)
• Has a family member who is a suitable stem cell donor

Diseases Treated:

Severe combined immunodeficiency (SCID)

Eligibility:

• Infant at least 2 months old with severe combined immunodeficiency (SCID)
• Has suitable matched sibling donor or matched unrelated donor or a single haplotype matched family member donor

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

• Children and young adults up to 21 years old
• Refractory or relapsed CD19+ acute lymphoblastic leukemia (ALL)

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial open only to St. Jude patients.

• 12 years old or younger
• Has had a hematopoietic stem cell transplant (HCST)
• Recruitment occurs within two weeks before or after discharge from transplant admission
• Prescribed oral medication at discharge
• Speaks and reads English

Diseases Treated:

Brain Tumors

Solid Tumors

Eligibility:

• 12 months old or older and younger than 21 years old
• Diagnosis:
  • Part A - Non-central nervous system (CNS) tumor that returned after treatment or did not respond to treatment
  • Part B – CNS tumor that returned after treatment or did not respond to treatment
• No other proven therapy options

Diseases Treated:

Brain Tumor : Previously treated brain tumors

Eligibility:

• Age is > 6 years and < 17 years & 10 months at the time of study entry.
• Off treatment and progression–free for at least 12 months and less than and equal to 14 years.
• Diagnosis of a primary brain tumor treated with at least one of the following :
  • Neurosurgical resection of the brain tumor;
  • Cranial irradiation;
  • Any chemotherapy to treat the brain tumor

Diseases Treated:

Solid Tumors, Central Nervous System Tumors

Eligibility:

Phase 1

• Birth through 21 years of age with locally advanced or metastatic solid tumor or primary CNS tumor that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy, OR
• Infants from birth and older with diagnosis of malignancy and with documented NTRK fusion that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy, OR
• Patients with locally advanced infantile fibrosarcoma who would require disfiguring surgery or limb amputation to achieve complete surgical resection

Phase 2

• Infants from birth and older at C1D1 with locally advanced or metastatic infantile fibrosarcoma OR
• Birth through 21 years old at C1D1 with locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was not responsive to therapy and for which no standard or available curative therapy with a NTRK gene funsion OR
• More than 21 years old with tumor diagnosis typical of a pediatric patient and an NTRK fusion

Diseases Treated:

Relapsed or refractory solid tumor

Eligibility:

• Participant is at least one month old and younger than 18 years old at the time of signing the informed consent (in Part A, participants < 2 years of age are not included).
• Participant has a histologically confirmed solid tumor
• Participant has a disease that is relapsed or refractory to all potentially curative standard treatment regimens, or has a disease for which there is no standard treatment regimen that is potentially curative.

Diseases Treated:

Brain Tumors

Eligibility:

• Between 6 and 21 years old
• Diagnosis of localized low-grade glioma, craniopharyngioma, ependymoma or germ cell tumor 
• Initiating focal cranial radiation therapy (photon or proton)
• Able to swallow pills
• Participant and parent/legal guardian speak, read and understand English
• Normal ECG and laboratory tests
• Adequate vision and hearing

Diseases Treated:

Gliomas

Eligibility:

• Participant is ≥ 3 but ≤ 21 years of age at registration.
• Participant has a diagnosis of low grade glioma (Grades I & II) or optic pathway gliomas with clinical and/or radiographic evidence of progression.
• Participant received last fraction of local irradiation to the primary tumor ≥ 12 weeks prior to registration, or does not apply.

Diseases Treated:

Retinoblastoma protein 1 (Rb1) positive recurrent, progressive, or refractory central nervous system tumors

Eligibility:

• Between 4 and 21 years old
• Able to swallow capsules
• Rb1-positive recurrent, progressive or refractory central nervous system (CNS) tumor or DIPG
• Stable neurological deficit for at least 1 week prior to enrollment
• Last dose of known myelosuppressive anticancer chemotherapy at least 3 weeks prior to enrollment
• Last fraction of focal irradiation > 2 weeks prior to enrollment

Diseases Treated:

Recurrent, progressive, or refractory high-grade gliomas

Eligibility:

• Participant has recurrent, progressive or refractory non-brainstem high-grade glioma or diffuse intrinsic pontine glioma (DIPG)
• Participant is 1 to 18 years of age
• Participant has bi-measurable disease on MRI 
• Participant has received prior radiation therapy and/or chemotherapy and has not received myelosuppressive anticancer chemotherapy for at least three (3) weeks

Diseases Treated:

Diffuse Intrinsic Pontine Glioma (DIPG)

Eligibility:

• Diagnosis of progressive diffuse intrinsic pontine glioma (DIPG) or an increase in the bi-dimensional measurement or the appearance of a new tumor lesion since diagnosis
• At least 2 but not more than 22 years old
• Able to swallow capsules whole

Diseases Treated:

High-grade glioma (HGG), ependymoma

Eligibility:

• Between 5 and 21 years old
• Diagnosis of supratentorial high-grade glioma (HGG) or supratentorial ependymoma that is recurrent, progressive or refractory
• Head circumference of at least 44 cm
• Failed standard therapy with no other available treatment options
• Recovered from prior chemotherapy, immunotherapy or radiotherapy
• Willing and able to use the device at least 18 hours a day for at least 23 days and keep head shaved throughout treatment

Diseases Treated:

Recurrent or progressive low grade gliomas (brain tumors)

Eligibility:

• Participant is three (3) to twenty-one (21) years of age.
• Patient has refractory, progressive or recurrent confirmed low-grade glioma (WHO grade I or II) that was confirmed histologically at initial diagnosis.
• Participant received his or her last dose of myelosuppressive anticancer chemotherapy at least three (3) weeks prior to study registration, or at least six (6) weeks if nitrosourea.
• Participant received his or her last dose of other investigational or biological agent more than seven (7) days prior to study entry.

Diseases Treated:

Brain tumors (recurrent and refractory)

Eligibility:

• 25 years or younger at time of registration
• Histologically confirmed diagnosis of a primary central nervous system tumor
• Positive test for the BRAF^V600E or the BRAF Ins T mutation at a CLIA-approved laboratory

Diseases Treated:

DIPG, glioma

Eligibility:

• Newly diagnosed diffuse intrinsic pontine glioma (DIPG) or other glioma
• At least 3 years old and 21 or younger
• Positive for HLA-A2
• Has undergone tumor biopsy
• Positive H3.3 K27M mutation in tumor
• Has received standard radiation therapy
• No prior chemotherapy, immunotherapy or bone marrow transplant

Diseases Treated:

Recurrent ependymoma

Eligibility:

• Progressive intracranial ependymoma after prior focal irradiation
• At least 1 year old and younger than 21 years old
• Adequate performance status (ECOG less than 3)
• Does not require mechanical ventilation
• Interval from start of initial radiation therapy to enrollment greater than 9 months

Diseases Treated:

Craniopharyngioma

Eligibility:

• 21 years old or younger
• Diagnosis of craniopharyngioma

Diseases Treated:

Brain Tumor : Recurrent, progressive, or newly diagnosed atypical teratoid rhabdoid brain tumors (ATRT)
Solid Tumor : Progressive extra-CNS malignant rhabdoid solid tumors (MRT)

Eligibility:

• Has one of these types of tumors:
  • Newly diagnosed atypical teratoid rhabdoid tumors (ATRTs) or synchronous extraneural ATRTs
  • ATRTs or malignant rhabdoid tumors (MRTs) that have come back after previous treatment (recurrent disease)
  • ATRTs or MRTs that are growing after previous treatment (progressive disease)
• 21 years of age or younger

Diseases Treated:

Brain tumor, medulloblastoma, ependymoma, high-grade glioma (HGG), CNS embryonal tumors, and typical teratoid rhabdoid tumor (ATRT)

Eligibility:

Ages for Study Enrollment

• Stratum A (ribociclib and gemcitabine):  Between 1 and 24 years old with recurrent, progressive or refractory non-WNT,  non-sonic hedgehog (SHH) (NWNS) medulloblastoma or ependymoma
• Stratum B (ribociclib and trametinib):  Between 1 and 24 years old with recurrent, progressive, or refractory central nervous system (CNS) tumors, including:
  • High-grade glioma
  • Atypical teratoid rhabdoid tumor (ATRT)
  • SHH and WNT medulloblastoma
  • CNS embryonal tumors [previously called PNET])
• Stratum C (ribociclib and sonidegib):  Between 10 and 39 years old with recurrent, progressive, or refractory SHH medulloblastoma and copy number loss of 9q or PTCH1 mutation. (This stratum is only open to patients with SHH medulloblastoma who are fully grown.  Patients younger than 18 years old will have bone age to determine if fully grown)

Screening Phase Eligibility

Inclusion Criteria:

• Participants with recurrent, progressive, or refractory brain tumors
• At least 1 year old and younger than 25 years old at the time of screening. Exception: Participants with recurrent, progressive or refractory medulloblastoma who are at least 1 year old and younger than 40 years of age at the time of study screening are eligible for screening.
• Participants and/or guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines.

Exclusion Criteria:

• Recurrent, progressive or refractory low grade glioma (LGG)
• Prior exposure to a CDK4/6 inhibitor
• History of clinically significant, uncontrolled heart disease and/or repolarization abnormalities
• History of QTc prolongation

Diseases Treated:

Medulloblastoma (includes all variants of medulloblastoma and posterior fossa PNET)

Eligibility:

• Diagnosis of newly medulloblastoma
• At least 3 years old and younger than 22 years old (Strata W, S or N) OR
• At least 22 years old and younger than 40 years old AND has SHH medulloblastoma (Stratum S)
• Has not received previous treatment with radiation therapy or chemotherapy
• Must start treatment within 36 days of surgery to remove the tumor

Diseases Treated:

DIPG, diffuse midline glioma, H3 K27M-mutant glioma

Eligibility:

• Between 2 and 22 years old
• Diagnosed with diffuse intrinsic pontine glioma (DIPG) or other midline glioma that has not spread
• No prior therapy, other than surgery and/or steroids

Diseases Treated:

Brain tumor

Eligibility:

This study is open only to patients at St. Jude Children’s Research Hospital.

• Diagnosis of medulloblastoma or other brain tumor
• Between 8 and 12 years old
• At least 2 years after completion of therapy
• Full-time student
• Speaks English

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial that is open to St. Jude patients.

• Solid tumor or liquid tumor (cancerous or non-cancerous)

Eligibility:

• Diagnosis of a primary immunodeficiency predisposed to hemophagocytic lymphohistiocytosis
• Meets at least 4 of 8 diagnostic criteria for HLH as defined by the Histiocyte Society and is receiving HLH-directed therapy
• High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy

Eligibility:

This is a non-therapeutic clinical trial open to St. Jude patients only.

Participants have been pre-identified by the primary care team as an English-speaking, four-member family unit diagnosed with TP53 mutation.

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients

Diseases Treated:

None

Eligibility:

This is a non-therapeutic clinical trial that is open only to St. Jude patients.

• Younger than 25 years
• Undergoing cancer therapy at St. Jude Children’s Research Hospital
• Considered to be at high risk of infection

Diseases Treated:

Non-therapeutic clinical trial

Eligibility:

This is a non-therapeutic clinical trial that is open to children and adults with familial cancer and to their eligible family members.

Note: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical genetic testing, please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for the SJFAMILY study, as outlined below, you may enroll regardless of the results of your clinical genetic testing.

In this research study, the definition of “Familial Cancer” is met if any of the following is present:

• An individual with a history of cancer diagnosed under 26 years of age who has at least one first, second or third degree relative with a history of cancer diagnosed under 51 years of age; OR
• An individual who has been diagnosed  with more than one cancer, at least one of which was diagnosed under 26 years of age; OR
• An individual with a clinical or molecular diagnosis of a known cancer predisposition syndrome; OR
• An individual with a congenital cancer that was diagnosed before 6 months of age; OR
• An individual with a rare pediatric cancer or tumor diagnosed before 26 years of age. Examples of rare pediatric tumors include (but are not limited to): carcinoma or precursor carcinoma lesions of any organ or anatomic site and cutaneous malignant cancers. Additional rare pediatric tumors to be included will be defined as any solid malignancy or borderline tumor characterized by an annual incidence of less than two occurrences per million per year (such as ectomesenchymoma). 

Eligible individuals include:

• An individual who meets the definition of “Familial Cancer,” as above
• Blood relatives of the above individual with familial cancer, who are affected or unaffected by cancer

Diseases Treated:

Malaria

Eligibility:

• Healthy male or female adults
• Age 18 to 55 years old
• Minimum weight of 50 kg with BMI between 18 and 34 kg/m^2
• Females must be post-menopausal or have documented hysterectomy or bilateral oophorectomy

Eligibility:

• Child is greater than or equal to twelve (12) months and less than twenty-four (24) months.
• Child has adequate blood, liver and kidney function.
• Child has no history of lung disease, asthma, and hospitalization for respiratory illness, immunodeficiency, sickle cell disease, or any other serious underlying condition.

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

St. Jude patients:

• Treated with an allogeneic bone marrow or stem cell transplant at St. Jude
• 8 years of age or older
• Younger than 21 at time of transplant
• More than 5 years from date of transplant

Healthy volunteers (comparison group):

• 8 years of age or older
• Primarily speaks English
• No known history of serious illness

Eligibility:

• Diagnosis of a primary immunodeficiency predisposed to hemophagocytic lymphohistiocytosis
• Meets at least 4 of 8 diagnostic criteria for HLH as defined by the Histiocyte Society and is receiving HLH-directed therapy
• High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy

Diseases Treated:

Langerhans cell histiocytosis and LCH-related disorders

Eligibility:

• Prior diagnosis of Langerhans cell histiocytosis or LCH-related disorder
• Evidence of active disease
• Performance Score > 70% (use Lansky score for age < 16 and Karnofsky score for age ≥16)
• Any age
• Adequate organ functions

Diseases Treated:

Langerhans cell histiocytosis

Eligibility:

• Diagnosis of Langerhans cell histiocytosis
• Younger than 18 years old
• Meets inclusion criteria for the respective stratum

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant received an integrating retroviral or lentiviral vector based gene transfer or gene marked product at St. Jude Children’s Research Hospital within the past 15 year time period.

Eligibility:

• Diagnosis of a primary immunodeficiency predisposed to hemophagocytic lymphohistiocytosis
• Meets at least 4 of 8 diagnostic criteria for HLH as defined by the Histiocyte Society and is receiving HLH-directed therapy
• High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy

Diseases Treated:

X-linked Severe Combined Immunodeficiency (SCID-X1)

Eligibility:

• Diagnosis of Severe Combined Immunodeficiency, X-linked (SCID-X1)
• Newborn to 2 years of age
• No prior therapy with allogeneic stem cell transplantation
• No HIV infection

Diseases Treated:

Severe combined immunodeficiency (SCID)

Eligibility:

• Infant at least 2 months old with severe combined immunodeficiency (SCID)
• Has suitable matched sibling donor or matched unrelated donor or a single haplotype matched family member donor

Diseases Treated:

HIV Infection

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• The subject has been enrolled on IMPAACT P1025.

Diseases Treated:

Malaria

Eligibility:

• Healthy male or female adults
• Age 18 to 55 years old
• Minimum weight of 50 kg with BMI between 18 and 34 kg/m^2
• Females must be post-menopausal or have documented hysterectomy or bilateral oophorectomy

Diseases Treated:

Cancer or allogeneic hematopoietic cell transplantation

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant > 2 months of age and < 21 years of age at enrollment.
• Participant has or is scheduled to have an external tunneled central venous catheter (CVC) (Broviacs, Hickmans, tunneled percutaneously inserted central PICCs), that is expected to remain in place for an additional > 3 months.
• Participant with a current or previous line infection completed antibiotics > 14 days ago.

Diseases Treated:

Infectious Disease

Eligibility:

• 2 months old or older
• Diagnosed with adenovirus (AdV) infection
• Has symptoms related to AdV infection
• Able to take and absorb oral medication
• No previous treatment with brincidofovir (BCV)

Diseases Treated:

HIV Infection

Eligibility:

• Participant does not have life expectancy of less than 1 year.
• Participant does not have history of significant drug sensitivity or drug allergy.
• Participant is not participating in any other clinical trial without prior approval from sponsor while participating in this trial.
• Participant does not have an ongoing serious infection requiring systemic antibiotic therapy at the time of screening.

Diseases Treated:

Influenza

Eligibility:

• At least 3 years old and younger than 17
• Allogeneic hematopoietic stem cell transplant recipient
• At least 3 and not more than 35 months after transplant
• Platelet count of at least 30,000

Diseases Treated:

HIV/AIDS

Eligibility:

• Participant is HIV-1 infected male or female aged six (6) to less than eight-teen (18) years of age at Baseline.
• Participant is currently on a stable 2-NRTI (TDF containing) regimen that includes a protocol specified 3rd ARV agent for greater than or equal to six (6) consecutive months prior to screening.
• Participant does not have an acquired immunodeficiency syndrome (AIDS) indicator condition with onset within thirty (30) days prior to screening.

Diseases Treated:

Depression

Eligibility:

• 12 to 24 years old
• Receiving mental health or HIV-related care at participating IMPAACT site
• HIV-positive
• Diagnosis of nonpsychotic depression
• Current depressive symptoms that warrant intervention
• Reads and speaks English

Diseases Treated:

Non-Therapeutic

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Between 4 and 21 years old
• Newly confirmed diagnosis of acute myeloid leukemia (AML) or scheduled to receive conditioning for allogeneic hematopoietic stem cell transplantation within seven days
• Receiving treatment at St. Jude Children’s Research Hospital

Diseases Treated:

Human Immunodeficiency Virus

Eligibility:

• At least 4 weeks old but younger than 18 years old
• Confirmed HIV-1 infection
• Current or past treatment with HIV medications (Children younger than 2 years old may be eligible even if they have not been treated with HIV medications.)

Diseases Treated:

None

Eligibility:

This is a non-therapeutic clinical trial that is open only to St. Jude patients.

• Younger than 25 years
• Undergoing cancer therapy at St. Jude Children’s Research Hospital
• Considered to be at high risk of infection

Eligibility:

• Child is greater than or equal to twelve (12) months and less than twenty-four (24) months.
• Child has adequate blood, liver and kidney function.
• Child has no history of lung disease, asthma, and hospitalization for respiratory illness, immunodeficiency, sickle cell disease, or any other serious underlying condition.

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

This is a non-therapeutic clinical trial open to patients receiving treatment at a Children’s Oncology Group (COG)-affiliated institution.

• At least 8 years old and younger than 16 years old
• Newly diagnosed acute lymphoblastic leukemia (ALL), in first remission
• Completed chemotherapy within past 6 months
• Performance status corresponding to ECOG scores of 0, 1, 2
• Fewer than 420 minutes of moderate to vigorous physical activity (MVPA) over the last week
• Access to smart phone (Android 4.3 or later, iOS 7.1 or later or computer with connection to the internet)
• Able to write and read English (patient and at least one parent/guardian)
• Not pregnant

Diseases Treated:

Acute myeloid leukemia (AML)

Eligibility:

• Diagnosis of one of the following:
  • Acute myeloid leukemia (AML)
  • 5% to 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality
  • Myeloid sarcoma
  • High grade myelodysplastic syndrome (MDS) with greater than 5% blasts
  • Treatment-related myeloid neoplasms, including AML and MDS
• 28 days to 21 years old
• No prior therapy, except for one dose of intrathecal therapy and the use of hydroxyurea or low-dose cytarabine
• Not pregnant

Diseases Treated:

Leukemia

Eligibility:

This is a non-therapeutic clinical trial that is open only to patients who were treated at St. Jude Children’s Research Hospital since 1980 and are currently followed at the hospital.

• St. Jude Children’s Research Hospital patient with acute leukemia (i.e., ALL or AML)
• Trisomy 21 Down syndrome diagnosis
• Completed all cancer therapy at St. Jude since 1980 and at least six months prior to study visit
• English as the primary language

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial that is open to St. Jude patients.

• Solid tumor or liquid tumor (cancerous or non-cancerous)

Diseases Treated:

• Acute lymphoblastic leukemia
• Acute myeloid leukemia
• Myelodysplasia
• Hodgkin lymphoma
• Non-Hodgkin lymphoma
• Juvenile myelomonocytic leukemia

Eligibility:

For transplant recipient:

• 21 years or younger
• Does not have a suitable sibling donor or volunteer unrelated donor
• Has a suitable single haplotype matched family member donor
• Diagnosed with high risk hematologic malignancy
• No prior allogeneic hematopoietic cell transplant

Diseases Treated:

Leukemia and lymphoma

Eligibility:

• 21 years and younger
• Does not have a suitable HLA-matched sibling donor or volunteer HLA-matched unrelated donor or is not a candidate for conventional matched donor transplant due to refractory disease
• Has a suitable single haplotype-matched family member donor
• High-risk hematologic malignancy, including certain diagnoses of:
  • Acute lymphoblastic leukemia (ALL)
  • Acute myeloid leukemia (AML)
  • Chronic myelogenous leukemia (CML)
  • Myelodysplastic syndrome (MDS)
  • Hodgkin lymphoma
  • Non-Hodgkin lymphoma

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

• Younger than 22 years old
• Diagnosis of B-cell acute lymphoblastic leukemia (B-ALL) with minimal residual disease (MRD) between 0.1 and 4.99% after prior chemotherapy, relapse or stem cell transplant

Diseases Treated:

Acute lymphoblastic leukemia, lymphoblastic lymphoma

Eligibility:

• Acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LL) that has not responded to treatment or has come back after treatment
• Between 4 and 45 years old
• Weigh at least 20 kg (44 lbs)
• Able to swallow pills

Diseases Treated:

Leukemia and other blood diseases

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant is receiving an allogeneic hematopoietic stem cell (HSC) transplant at St. Jude Children's Research Hospital using an unlicensed cord blood unit (CBU).
• Participant may be of any age and either gender.
• Participant has a medical disorder affecting the hematopoietic system that is inherited, acquired, or a result from myeloablative treatment.
• Participant is receiving HPC-CORD BLOOD product manufactured by NCBP (at least one, if the graft contains more than one units).

Diseases Treated:

• Leukemia
• Lymphoma
• Myeloid sarcoma
• Myelodysplastic syndrome

Eligibility:

• 21 years old and younger
• Diagnosed with one of the following that has come back or did not improve after bone marrow transplant
  • Acute lymphoblastic leukemia (ALL)
  • Acute myeloid leukemia
  • Myeloid sarcoma
  • Chronic myeloid leukemia (CML)
  • Juvenile myelomonocytic leukemia (JMML)
  • Myelodysplastic syndrome (MDS)
  • Non-Hodgkin lymphoma (NHL)
• Has a family member who is a suitable stem cell donor

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

• Younger than 22 years old
• Diagnosis of relapsed or refractory acute lymphoblastic leukemia (ALL)

Diseases Treated:

Relapsed or refractory leukemia or hematologic malignancies

Eligibility:

(Phase II)

• 21 years of age or younger
• Acute myeloid leukemia (AML)
• No history of HIV infection

Diseases Treated:

Leukemia and lymphoma

Eligibility:

• 21 years of age or younger
• Newly diagnosed mature B-cell non-Hodgkin lymphoma and leukemia
• No previous treatment (no more than 72 hours of steroids, one intrathecal chemotherapy treatment, and/or emergency radiation)

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

• Children and young adults up to 21 years old
• Refractory or relapsed CD19+ acute lymphoblastic leukemia (ALL)

Diseases Treated:

Acute lymphoblastic leukemia (ALL)

Eligibility:

• Newly diagnosed ALL
• 1 year of age or younger at the time of diagnosis
• Has not had any — or has had limited — prior therapy
• Does not have mature B-cell ALL, acute myeloid leukemia (AML) or Down syndrome

Diseases Treated:

Leukemia and lymphoma

Eligibility:

• Diagnosis of B-cell or T-cell acute lymphoblastic leukemia (ALL) or acute lymphoblastic lymphoma (LLy)
• 1 to 18 years old
• No prior therapy or limited prior therapy

Diseases Treated:

Acute myeloid leukemia (AML), acute undifferentiated leukemia, mixed phenotype acute leukemia

Eligibility:

• Diagnosis of relapsed or refractory acute myeloid leukemia (AML), acute undifferentiated leukemia or mixed phenotype acute leukemia 
• At least 2 years old and younger than 22 years old
• Recovered from acute effects of prior therapy and no evidence of graft-versus-host disease (GVHD)
• Adequate liver, kidney and heart functions

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

Childhood cancer incidence and survival

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Newly diagnosed with cancer between January 1, 1970, and December 31, 1996
• Survival five years from diagnosis
• Age less than 21 years at the time of diagnosis of cancer
• English or Spanish speaking and living in the United States or Canada at the time of diagnosis

Eligibility:

This is a non-therapeutic clinical trial open to St. Jude patients only.

Participant is a St. Jude Life or ACT (After Completion of Therapy) patient.

Diseases Treated:

For survivors of Hodgkin lymphoma

Eligibility:

• Females, 25 years of age or older.
• Participant does not plan to become pregnant in the next 2 years and is not currently breast feeding.

Diseases Treated:

Cancer

Eligibility:

• Younger than 21 years old at time of cancer diagnosis
• Weigh ≥ 40 Kg
• Lifetime cumulative anthracycline dose prior to age 22: total dose ≥ 250 mg/m² DOXOrubicin equivalent without the protection of dexrazoxane (Zinecard) therapy
• Completed cancer treatment at least 2 years prior to study enrollment

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant was enrolled on SJCRH TOTXV ALL protocol (or Best Clinical Management that followed the same treatment provided in the TOTXV protocol)
• Minimum of five years post diagnosis of ALL
• Minimum age of 8.0 years at time of follow-up evaluation

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant has a diagnosis of osteonecrosis and a hematologic malignancy or sickle cell disease.
• Participant is a candidate for hip core decompression surgery.
• Participant is 8 to 29 years of age.

Diseases Treated:

Congestive heart failure

Eligibility:

• Participant is > 16 years of age.
• Participant had cancer diagnosis < 21 years of age.
• Participant has lifetime cumulative anthracycline dose > 300 mg/m2.
• Participant is > 2 years since completion of treatment for cancer.

Diseases Treated:

Solid tumors

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Please see clinical trial for full inclusion and exclusion eligibility criteria.

Diseases Treated:

Long-term effects of childhood cancer

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Currently enrolled on the SJLIFE protocol
• 18 to 49 years old
• At least 10 years post first cancer diagnosis
• Lives within a 45-minute drive of a Greater Memphis Area ATC Fitness Center
• Speaks English
• Low lean mass defined as either:
  • Body fat content of at least 25% in males or at least 35% in females OR
  • Age and sex-specific Relative Lean Muscle Mass Standard Deviation score <-1.0

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant was diagnosed with a childhood cancer which was treated or followed by St. Jude Children's Research Hospital
• Participant completed treatment for disease at least ten years prior to entry in this study
• Participant is willing to return to St. Jude for periodic evaluation

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• All patients being followed or treated at St. Jude Children’s Research Hospital for the diagnosis of childhood cancer
• Patient has not been permanently discharged from care and follow-up

Diseases Treated:

Hodgkin lymphoma

Eligibility:

This is a non-therapeutic clinical trial that is only open to former St. Jude patients, their family members and friends.

Hodgkin lymphoma survivor

• Current St. Jude LIFE participant, treated with thoracic radiation for Hodgkin lymphoma
• At least 18 years old
• At least 5 years from original diagnosis

Comparison group

• Sibling, parent, relative or friend of a current or former St. Jude patient
• At least 18 years old

Diseases Treated:

Lymphoma

Eligibility:

• Previously untreated CD30+ classical Hodgkin lymphoma
• 21 years or younger at time of diagnosis (low-risk and intermediate-risk patients)
• 18 years or younger (high-risk patients)

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial that is open to St. Jude patients.

• Solid tumor or liquid tumor (cancerous or non-cancerous)

Diseases Treated:

Leukemia and lymphoma

Eligibility:

• 21 years and younger
• Does not have a suitable HLA-matched sibling donor or volunteer HLA-matched unrelated donor or is not a candidate for conventional matched donor transplant due to refractory disease
• Has a suitable single haplotype-matched family member donor
• High-risk hematologic malignancy, including certain diagnoses of:
  • Acute lymphoblastic leukemia (ALL)
  • Acute myeloid leukemia (AML)
  • Chronic myelogenous leukemia (CML)
  • Myelodysplastic syndrome (MDS)
  • Hodgkin lymphoma
  • Non-Hodgkin lymphoma

Diseases Treated:

For survivors of Hodgkin lymphoma

Eligibility:

• Females, 25 years of age or older.
• Participant does not plan to become pregnant in the next 2 years and is not currently breast feeding.

Diseases Treated:

• Leukemia
• Lymphoma
• Myeloid sarcoma
• Myelodysplastic syndrome

Eligibility:

• 21 years old and younger
• Diagnosed with one of the following that has come back or did not improve after bone marrow transplant
  • Acute lymphoblastic leukemia (ALL)
  • Acute myeloid leukemia
  • Myeloid sarcoma
  • Chronic myeloid leukemia (CML)
  • Juvenile myelomonocytic leukemia (JMML)
  • Myelodysplastic syndrome (MDS)
  • Non-Hodgkin lymphoma (NHL)
• Has a family member who is a suitable stem cell donor

Diseases Treated:

Leukemia and lymphoma

Eligibility:

• 21 years of age or younger
• Newly diagnosed mature B-cell non-Hodgkin lymphoma and leukemia
• No previous treatment (no more than 72 hours of steroids, one intrathecal chemotherapy treatment, and/or emergency radiation)

Diseases Treated:

Leukemia and lymphoma

Eligibility:

• Diagnosis of B-cell or T-cell acute lymphoblastic leukemia (ALL) or acute lymphoblastic lymphoma (LLy)
• 1 to 18 years old
• No prior therapy or limited prior therapy

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Please see the eligibility section on the clinical trial for full inclusion and exclusion criteria.

Diseases Treated:

Relapsed or refractory solid tumors

Eligibility:

• Participant is >12 months and < 21 years of age at the time of study entry.
• Participant has had histologic verification of malignancy at original diagnosis or relapse.
• Participant has serum tumor markers including alpha-fetoprotein or beta-HCG.
• Participant has a relapsed or refractory solid tumor.

Diseases Treated:

Solid Tumors

Eligibility:

• Diagnosis of solid tumor that is resistant to other anticancer therapy or has come back after therapy. Includes lymphoma and central nervous system (CNS) tumors that may or may not require surgery.
• Fully recovered from the acute toxic effects of all prior anticancer therapy
• At least 1 but not more than 21 years of age
• Able to swallow tablets whole

Diseases Treated:

Brain Tumors

Solid Tumors

Eligibility:

• 12 months old or older and younger than 21 years old
• Diagnosis:
  • Part A - Non-central nervous system (CNS) tumor that returned after treatment or did not respond to treatment
  • Part B – CNS tumor that returned after treatment or did not respond to treatment
• No other proven therapy options

Diseases Treated:

Brain Tumor : Previously treated brain tumors

Eligibility:

• Age is > 6 years and < 17 years & 10 months at the time of study entry.
• Off treatment and progression–free for at least 12 months and less than and equal to 14 years.
• Diagnosis of a primary brain tumor treated with at least one of the following :
  • Neurosurgical resection of the brain tumor;
  • Cranial irradiation;
  • Any chemotherapy to treat the brain tumor

Diseases Treated:

For patients at risk for blood clots

Eligibility:

• Participant has a diagnosis requiring anticoagulant therapy.
• Participant has completed their standard anticoagulant therapy.
• Participant is not receiving active therapy for a malignant condition.
• Participant is less than 18 years of age at the time of consent.
• Participant and legal guardian agree to food and drug restrictions during study.

Diseases Treated:

HIV Infection

Eligibility:

• Participant does not have life expectancy of less than 1 year.
• Participant does not have history of significant drug sensitivity or drug allergy.
• Participant is not participating in any other clinical trial without prior approval from sponsor while participating in this trial.
• Participant does not have an ongoing serious infection requiring systemic antibiotic therapy at the time of screening.

Diseases Treated:

Hemophilia B

Eligibility:

• Male
• At least 18 years old
• Diagnosis of hemophilia B

Diseases Treated:

Neuroblastoma, pheochromocytoma

Eligibility:

This study is open to St. Jude patients only.

• Known or suspected neuroblastoma or pheochromocytoma
• At least 1 year old

Diseases Treated:

Lymphocyte predominant Hodgkin lymphoma 

Eligibility:

• Diagnosis of early-stage lymphocyte-predominant Hodgkin lymphoma (LPHL)
• LPHL that has not been previously treated with chemotherapy or radiation therapy
• 18 years of age or younger

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

• Younger than 22 years old
• Diagnosis of B-cell acute lymphoblastic leukemia (B-ALL) with minimal residual disease (MRD) between 0.1 and 4.99% after prior chemotherapy, relapse or stem cell transplant

Diseases Treated:

Retinoblastoma

Eligibility:

• Age 17 or younger
• Diagnosis of retinoblastoma that did not go away with treatment or came back after treatment

Diseases Treated:

For survivors of Hodgkin lymphoma

Eligibility:

• Females, 25 years of age or older.
• Participant does not plan to become pregnant in the next 2 years and is not currently breast feeding.

Diseases Treated:

Solid Tumors, Central Nervous System Tumors

Eligibility:

Phase 1

• Birth through 21 years of age with locally advanced or metastatic solid tumor or primary CNS tumor that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy, OR
• Infants from birth and older with diagnosis of malignancy and with documented NTRK fusion that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy, OR
• Patients with locally advanced infantile fibrosarcoma who would require disfiguring surgery or limb amputation to achieve complete surgical resection

Phase 2

• Infants from birth and older at C1D1 with locally advanced or metastatic infantile fibrosarcoma OR
• Birth through 21 years old at C1D1 with locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was not responsive to therapy and for which no standard or available curative therapy with a NTRK gene funsion OR
• More than 21 years old with tumor diagnosis typical of a pediatric patient and an NTRK fusion

Diseases Treated:

Cancer

Eligibility:

• Younger than 21 years old at time of cancer diagnosis
• Weigh ≥ 40 Kg
• Lifetime cumulative anthracycline dose prior to age 22: total dose ≥ 250 mg/m² DOXOrubicin equivalent without the protection of dexrazoxane (Zinecard) therapy
• Completed cancer treatment at least 2 years prior to study enrollment

Diseases Treated:

Relapsed or refractory solid tumor

Eligibility:

• Participant is at least one month old and younger than 18 years old at the time of signing the informed consent (in Part A, participants < 2 years of age are not included).
• Participant has a histologically confirmed solid tumor
• Participant has a disease that is relapsed or refractory to all potentially curative standard treatment regimens, or has a disease for which there is no standard treatment regimen that is potentially curative.

Diseases Treated:

Advanced melanoma or PD-L1 positive advanced relapsed or refractory solid tumor or lymphoma

Eligibility:

• Between 6 months and 18 years old with diagnosis of MSI-H solid tumor OR
• Between 12 years and 18 years old with diagnosis of melanoma OR
• Between 3 and 18 years old with relapsed or refractory classical Hodgkin lymphoma
• Negative pregnancy test 72 hours prior to medication administration in participants of child-bearing potential
• Appropriate liver and kidney functions

Diseases Treated:

Brain Tumors

Eligibility:

• Between 6 and 21 years old
• Diagnosis of localized low-grade glioma, craniopharyngioma, ependymoma or germ cell tumor 
• Initiating focal cranial radiation therapy (photon or proton)
• Able to swallow pills
• Participant and parent/legal guardian speak, read and understand English
• Normal ECG and laboratory tests
• Adequate vision and hearing

Diseases Treated:

Neuroblastoma

Eligibility:

• Younger than 19 years old
• Newly diagnosed, advanced stage, high-risk neuroblastoma
• Histologic proof of neuroblastoma or positive bone marrow for tumor cells with increased urine catecholamine
• Adequate kidney and liver functions

Diseases Treated:

Human Immunodeficiency Virus

Eligibility:

• At least 4 weeks old but younger than 18 years old
• Confirmed HIV-1 infection
• Current or past treatment with HIV medications (Children younger than 2 years old may be eligible even if they have not been treated with HIV medications.)

Diseases Treated:

Gliomas

Eligibility:

• Participant is ≥ 3 but ≤ 21 years of age at registration.
• Participant has a diagnosis of low grade glioma (Grades I & II) or optic pathway gliomas with clinical and/or radiographic evidence of progression.
• Participant received last fraction of local irradiation to the primary tumor ≥ 12 weeks prior to registration, or does not apply.

Diseases Treated:

Retinoblastoma protein 1 (Rb1) positive recurrent, progressive, or refractory central nervous system tumors

Eligibility:

• Between 4 and 21 years old
• Able to swallow capsules
• Rb1-positive recurrent, progressive or refractory central nervous system (CNS) tumor or DIPG
• Stable neurological deficit for at least 1 week prior to enrollment
• Last dose of known myelosuppressive anticancer chemotherapy at least 3 weeks prior to enrollment
• Last fraction of focal irradiation > 2 weeks prior to enrollment

Diseases Treated:

Diffuse Intrinsic Pontine Glioma (DIPG)

Eligibility:

• Diagnosis of progressive diffuse intrinsic pontine glioma (DIPG) or an increase in the bi-dimensional measurement or the appearance of a new tumor lesion since diagnosis
• At least 2 but not more than 22 years old
• Able to swallow capsules whole

Diseases Treated:

Sickle cell disease

Eligibility:

This is a non-therapeutic clinical trial that is open only to patients at St. Jude or Methodist Healthcare.

• Between 6 and 25 years old
• Diagnosed with sickle cell disease
• Seeking care for acute vaso-occlusive crisis pain at St. Jude Children’s Research Hospital or Methodist Healthcare
• Speaks English

Diseases Treated:

Recurrent or progressive low grade gliomas (brain tumors)

Eligibility:

• Participant is three (3) to twenty-one (21) years of age.
• Patient has refractory, progressive or recurrent confirmed low-grade glioma (WHO grade I or II) that was confirmed histologically at initial diagnosis.
• Participant received his or her last dose of myelosuppressive anticancer chemotherapy at least three (3) weeks prior to study registration, or at least six (6) weeks if nitrosourea.
• Participant received his or her last dose of other investigational or biological agent more than seven (7) days prior to study entry.

Diseases Treated:

DIPG, glioma

Eligibility:

• Newly diagnosed diffuse intrinsic pontine glioma (DIPG) or other glioma
• At least 3 years old and 21 or younger
• Positive for HLA-A2
• Has undergone tumor biopsy
• Positive H3.3 K27M mutation in tumor
• Has received standard radiation therapy
• No prior chemotherapy, immunotherapy or bone marrow transplant

Diseases Treated:

Congestive heart failure

Eligibility:

• Participant is > 16 years of age.
• Participant had cancer diagnosis < 21 years of age.
• Participant has lifetime cumulative anthracycline dose > 300 mg/m2.
• Participant is > 2 years since completion of treatment for cancer.

Diseases Treated:

• Leukemia
• Lymphoma
• Myeloid sarcoma
• Myelodysplastic syndrome

Eligibility:

• 21 years old and younger
• Diagnosed with one of the following that has come back or did not improve after bone marrow transplant
  • Acute lymphoblastic leukemia (ALL)
  • Acute myeloid leukemia
  • Myeloid sarcoma
  • Chronic myeloid leukemia (CML)
  • Juvenile myelomonocytic leukemia (JMML)
  • Myelodysplastic syndrome (MDS)
  • Non-Hodgkin lymphoma (NHL)
• Has a family member who is a suitable stem cell donor

Diseases Treated:

Recurrent ependymoma

Eligibility:

• Progressive intracranial ependymoma after prior focal irradiation
• At least 1 year old and younger than 21 years old
• Adequate performance status (ECOG less than 3)
• Does not require mechanical ventilation
• Interval from start of initial radiation therapy to enrollment greater than 9 months

Diseases Treated:

Craniopharyngioma

Eligibility:

• 21 years old or younger
• Diagnosis of craniopharyngioma

Diseases Treated:

Metastatic sarcomas

Eligibility:

• Participant has histologically or cytologically confirmed diagnosis:
  • Nonrhabdomyosarcoma of soft tissue
  • Ewing sarcoma
  • Osteosarcoma at any site
• Participant must be greater than 3 years of age and < 40 years of age.
• Participant has not had any prior radiotherapy to the treatment site.

Diseases Treated:

Relapsed or refractory leukemia or hematologic malignancies

Eligibility:

(Phase II)

• 21 years of age or younger
• Acute myeloid leukemia (AML)
• No history of HIV infection

Eligibility:

• Child is greater than or equal to twelve (12) months and less than twenty-four (24) months.
• Child has adequate blood, liver and kidney function.
• Child has no history of lung disease, asthma, and hospitalization for respiratory illness, immunodeficiency, sickle cell disease, or any other serious underlying condition.

Diseases Treated:

Brain Tumor : Recurrent, progressive, or newly diagnosed atypical teratoid rhabdoid brain tumors (ATRT)
Solid Tumor : Progressive extra-CNS malignant rhabdoid solid tumors (MRT)

Eligibility:

• Has one of these types of tumors:
  • Newly diagnosed atypical teratoid rhabdoid tumors (ATRTs) or synchronous extraneural ATRTs
  • ATRTs or malignant rhabdoid tumors (MRTs) that have come back after previous treatment (recurrent disease)
  • ATRTs or MRTs that are growing after previous treatment (progressive disease)
• 21 years of age or younger

Diseases Treated:

Brain tumor, medulloblastoma, ependymoma, high-grade glioma (HGG), CNS embryonal tumors, and typical teratoid rhabdoid tumor (ATRT)

Eligibility:

Ages for Study Enrollment

• Stratum A (ribociclib and gemcitabine):  Between 1 and 24 years old with recurrent, progressive or refractory non-WNT,  non-sonic hedgehog (SHH) (NWNS) medulloblastoma or ependymoma
• Stratum B (ribociclib and trametinib):  Between 1 and 24 years old with recurrent, progressive, or refractory central nervous system (CNS) tumors, including:
  • High-grade glioma
  • Atypical teratoid rhabdoid tumor (ATRT)
  • SHH and WNT medulloblastoma
  • CNS embryonal tumors [previously called PNET])
• Stratum C (ribociclib and sonidegib):  Between 10 and 39 years old with recurrent, progressive, or refractory SHH medulloblastoma and copy number loss of 9q or PTCH1 mutation. (This stratum is only open to patients with SHH medulloblastoma who are fully grown.  Patients younger than 18 years old will have bone age to determine if fully grown)

Screening Phase Eligibility

Inclusion Criteria:

• Participants with recurrent, progressive, or refractory brain tumors
• At least 1 year old and younger than 25 years old at the time of screening. Exception: Participants with recurrent, progressive or refractory medulloblastoma who are at least 1 year old and younger than 40 years of age at the time of study screening are eligible for screening.
• Participants and/or guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines.

Exclusion Criteria:

• Recurrent, progressive or refractory low grade glioma (LGG)
• Prior exposure to a CDK4/6 inhibitor
• History of clinically significant, uncontrolled heart disease and/or repolarization abnormalities
• History of QTc prolongation

Diseases Treated:

Medulloblastoma (includes all variants of medulloblastoma and posterior fossa PNET)

Eligibility:

• Diagnosis of newly medulloblastoma
• At least 3 years old and younger than 22 years old (Strata W, S or N) OR
• At least 22 years old and younger than 40 years old AND has SHH medulloblastoma (Stratum S)
• Has not received previous treatment with radiation therapy or chemotherapy
• Must start treatment within 36 days of surgery to remove the tumor

Diseases Treated:

DIPG, diffuse midline glioma, H3 K27M-mutant glioma

Eligibility:

• Between 2 and 22 years old
• Diagnosed with diffuse intrinsic pontine glioma (DIPG) or other midline glioma that has not spread
• No prior therapy, other than surgery and/or steroids

Diseases Treated:

Retinoblastoma

Eligibility:

• Newly diagnosed retinoblastoma that has not spread beyond the eye
• Has not received previous treatment with chemotherapy or radiation therapy
• Patients who have been diagnosed with retinoblastoma in one eye who did not receive chemotherapy, but then developed retinoblastoma in the opposite eye

Diseases Treated:

Acute lymphoblastic leukemia (ALL)

Eligibility:

• Newly diagnosed ALL
• 1 year of age or younger at the time of diagnosis
• Has not had any — or has had limited — prior therapy
• Does not have mature B-cell ALL, acute myeloid leukemia (AML) or Down syndrome

Diseases Treated:

Non-therapeutic

Eligibility:

This clinical trial is open only to patients at the St. Jude Children’s Research Hospital Hematology Clinic.

• Diagnosis of sickle cell disease (SCD)
• At least 12 years old and younger than 18 years old
• On a stable hydroxyurea dose in pill formation for 6 months or longer
• Lives with legal guardian
• Able to return to clinic at 4-week intervals

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial open only to parents of St. Jude patients.

• Parent of a child who received a haploidentical or unrelated donor transplant at St. Jude Children’s Research Hospital between January 1, 2006 and December 31, 2015
• Speaks and reads English

Diseases Treated:

Depression

Eligibility:

• 12 to 24 years old
• Receiving mental health or HIV-related care at participating IMPAACT site
• HIV-positive
• Diagnosis of nonpsychotic depression
• Current depressive symptoms that warrant intervention
• Reads and speaks English

Diseases Treated:

Brain Tumors

Eligibility:

• Between 6 and 21 years old
• Diagnosis of localized low-grade glioma, craniopharyngioma, ependymoma or germ cell tumor 
• Initiating focal cranial radiation therapy (photon or proton)
• Able to swallow pills
• Participant and parent/legal guardian speak, read and understand English
• Normal ECG and laboratory tests
• Adequate vision and hearing

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Please see the eligibility section on the clinical trial for full inclusion and exclusion criteria.

Diseases Treated:

Sickle cell disease

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

Cancer

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients and their parents.

• 8 to 17.9 years old
• Cancer survivor who is off cancer therapy
• Parents and legal guardians of children to also enroll in the study
• Speaks English

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic research study open only to St. Jude patients, their parents and patients' teachers.

• Patient between 4 and 6 years old
• Primary diagnosis of brain tumor or non-CNS solid tumor
• 6 to 12 months since therapy was completed (+/- 1 month) OR more than 18 months since therapy completion
• Chemotherapy was part of tumor treatment
• Able to speak and read English

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial open only to St. Jude patients.

• 12 years old or younger
• Has had a hematopoietic stem cell transplant (HCST)
• Recruitment occurs within two weeks before or after discharge from transplant admission
• Prescribed oral medication at discharge
• Speaks and reads English

Diseases Treated:

Brain tumor

Eligibility:

This study is open only to patients at St. Jude Children’s Research Hospital.

• Diagnosis of medulloblastoma or other brain tumor
• Between 8 and 12 years old
• At least 2 years after completion of therapy
• Full-time student
• Speaks English

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

St. Jude patients:

• Treated with an allogeneic bone marrow or stem cell transplant at St. Jude
• 8 years of age or older
• Younger than 21 at time of transplant
• More than 5 years from date of transplant

Healthy volunteers (comparison group):

• 8 years of age or older
• Primarily speaks English
• No known history of serious illness

Diseases Treated:

Cancer

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients and their parents.

• St. Jude childhood cancer survivors between 8 and 17 years old
• Parents of St. Jude childhood cancer survivors

Diseases Treated:

Cancer

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients and their parents.

• 8 to 17.9 years old
• Cancer survivor who is off cancer therapy
• Parents and legal guardians of children to also enroll in the study
• Speaks English

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

Non-therapeutic

Eligibility:

Primary oncologist:

• Attending physician age 18 years or older who provides medical care to patients in the Solid Tumor Clinic at St. Jude Children’s Research Hospital
• Primary hematology-oncology fellows age 18 or older (audio-recorded disease reevaluation only)

Parents of children with cancer:

• Age 18 or older
• Biological parent, step-parent or primary legal guardian
• Reads and speaks English

Patients:

• Primary oncologist is enrolled in the study.
• 30 years old or younger
• Diagnosed with a solid tumor and/or a brain tumor

Diseases Treated:

Solid tumors

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant is under the care of a St. Jude physician with a known or suspected, newly diagnosed bone or soft tissue sarcoma who will be treated on or as per disease specific protocols and has not begun therapy OR participant has started therapy and the needed research imaging can be performed within 2-5 days of starting treatment. 
• Participant has undergone or is scheduled for PET-CT, Bone Scan, or PET-CT and Bone Scan within two (2) weeks of the whole body and primary tumor DWI-MRI. 
• Participant meets institutional MRI safety screening requirements. 
• Participant has not undergone primary tumor resection prior to arrival to St. Jude. 

Diseases Treated:

Recurrent ependymoma

Eligibility:

• Progressive intracranial ependymoma after prior focal irradiation
• At least 1 year old and younger than 21 years old
• Adequate performance status (ECOG less than 3)
• Does not require mechanical ventilation
• Interval from start of initial radiation therapy to enrollment greater than 9 months

Diseases Treated:

Rhabdomyosarcoma (low-risk, intermediate-risk and high-risk)

Eligibility:

• Newly diagnosed rhabdomyosarcoma
• Younger than 22 years (eligible until 22nd birthday)
• No previous treatment with chemotherapy or radiation therapy

Diseases Treated:

Craniopharyngioma

Eligibility:

• 21 years old or younger
• Diagnosis of craniopharyngioma

Diseases Treated:

Metastatic sarcomas

Eligibility:

• Participant has histologically or cytologically confirmed diagnosis:
  • Nonrhabdomyosarcoma of soft tissue
  • Ewing sarcoma
  • Osteosarcoma at any site
• Participant must be greater than 3 years of age and < 40 years of age.
• Participant has not had any prior radiotherapy to the treatment site.

Diseases Treated:

Sickle cell anemia, thalassemia

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• At least 2 years old
• Diagnosed with sickle cell anemia or thalassemia
• No active disease, including painful crisis or active infection
• Adequate blood counts

Diseases Treated:

Non-therapeutic

Eligibility:

This clinical trial is open only to patients at the St. Jude Children’s Research Hospital Hematology Clinic.

• Diagnosis of sickle cell disease (SCD)
• At least 12 years old and younger than 18 years old
• On a stable hydroxyurea dose in pill formation for 6 months or longer
• Lives with legal guardian
• Able to return to clinic at 4-week intervals

Diseases Treated:

Non-malignant blood diseases (non-therapeutic)

Eligibility:

• Receiving therapy or a consultation for a non-malignant blood disorder
• Biological relatives (with or without a non-malignant blood disorder) who agree to undergo genetic testing

Diseases Treated:

Sickle Cell Disease

Eligibility:

This is a non-therapeutic study for patients at St. Jude Children’s Research Hospital and Methodist Adult Comprehensive Sickle Cell Center.

• Diagnosis of sickle cell disease (SCD)
• 16 to 20 years old
• English is primary language

Diseases Treated:

Sickle cell disease

Eligibility:

This is a non-therapeutic clinical trial that is open only to patients at St. Jude or Methodist Healthcare.

• Between 6 and 25 years old
• Diagnosed with sickle cell disease
• Seeking care for acute vaso-occlusive crisis pain at St. Jude Children’s Research Hospital or Methodist Healthcare
• Speaks English

Diseases Treated:

Sickle cell disease

Eligibility:

This study is limited to patients living in the Memphis, Tennessee region.

Phase I

• Diagnosis of sickle cell disease
• Between 15 and 44.9 years old
• Speaks English

Phase II

• Diagnosis of sickle cell disease
• Between 15 and 44.9 years old
• Receiving hydroxyurea treatment for sickle cell disease
• Owns a smart phone
• Not currently receiving text messages for improving hydroxyurea adherence
• Not currently undergoing hydroxyurea dose escalation
• Not on any investigational new drug intervention study for sickle cell disease

Diseases Treated:

Sickle cell disease

Eligibility:

• Participant has a diagnosis of Sickle Cell Disease of any genotype.

Diseases Treated:

Sickle cell disease

Eligibility:

• Participant with Sickle Cell Disease (laboratory determined HbSS or HbS beta zero thalassemia) who have had >2 episodes of Vaso-occlusive crisis in the past year. 
• Participants >2 years of age and < 18 years of age.

Diseases Treated:

Germ cell tumors

Eligibility:

• 14 years old or older
• Diagnosis of germ cell tumor (GCT)
• Previously received 3 to 6 cycles of cisplatin-based chemotherapy
• No more than one prior line of chemotherapy
• No prior treatment with high-dose chemotherapy

Diseases Treated:

Relapsed or refractory solid tumors

Eligibility:

• Participant is >12 months and < 21 years of age at the time of study entry.
• Participant has had histologic verification of malignancy at original diagnosis or relapse.
• Participant has serum tumor markers including alpha-fetoprotein or beta-HCG.
• Participant has a relapsed or refractory solid tumor.

Diseases Treated:

Solid Tumors

Eligibility:

• Diagnosis of solid tumor that is resistant to other anticancer therapy or has come back after therapy. Includes lymphoma and central nervous system (CNS) tumors that may or may not require surgery.
• Fully recovered from the acute toxic effects of all prior anticancer therapy
• At least 1 but not more than 21 years of age
• Able to swallow tablets whole

Diseases Treated:

Brain Tumors

Solid Tumors

Eligibility:

• 12 months old or older and younger than 21 years old
• Diagnosis:
  • Part A - Non-central nervous system (CNS) tumor that returned after treatment or did not respond to treatment
  • Part B – CNS tumor that returned after treatment or did not respond to treatment
• No other proven therapy options

Diseases Treated:

Neuroblastoma

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• All newly diagnosed suspected:
  • neuroblastoma,
  • ganglioneuroblastoma, or 
  • ganglioneuroma (maturing subtype) patients
• Less than or equal to 21 years of age
• Seen at Children's Oncology Group (COG) institutions

Diseases Treated:

Solid tumors

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• Participant is under the care of a St. Jude physician with a known or suspected, newly diagnosed bone or soft tissue sarcoma who will be treated on or as per disease specific protocols and has not begun therapy OR participant has started therapy and the needed research imaging can be performed within 2-5 days of starting treatment. 
• Participant has undergone or is scheduled for PET-CT, Bone Scan, or PET-CT and Bone Scan within two (2) weeks of the whole body and primary tumor DWI-MRI. 
• Participant meets institutional MRI safety screening requirements. 
• Participant has not undergone primary tumor resection prior to arrival to St. Jude. 

Diseases Treated:

Neuroblastoma, pheochromocytoma

Eligibility:

This study is open to St. Jude patients only.

• Known or suspected neuroblastoma or pheochromocytoma
• At least 1 year old

Diseases Treated:

Non-therapeutic

Eligibility:

This is a non-therapeutic clinical trial that is open to St. Jude patients.

• Solid tumor or liquid tumor (cancerous or non-cancerous)

Diseases Treated:

Adrenocortical tumors (carcinoma and adenoma)

Eligibility:

• Diagnosis of an adrenocortical tumor (ACT)
• 21 years of age or younger at diagnosis
• Relatives of patients with ACT

Diseases Treated:

Retinoblastoma

Eligibility:

• Age 17 or younger
• Diagnosis of retinoblastoma that did not go away with treatment or came back after treatment

Diseases Treated:

Solid Tumors, Central Nervous System Tumors

Eligibility:

Phase 1

• Birth through 21 years of age with locally advanced or metastatic solid tumor or primary CNS tumor that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy, OR
• Infants from birth and older with diagnosis of malignancy and with documented NTRK fusion that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy, OR
• Patients with locally advanced infantile fibrosarcoma who would require disfiguring surgery or limb amputation to achieve complete surgical resection

Phase 2

• Infants from birth and older at C1D1 with locally advanced or metastatic infantile fibrosarcoma OR
• Birth through 21 years old at C1D1 with locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was not responsive to therapy and for which no standard or available curative therapy with a NTRK gene funsion OR
• More than 21 years old with tumor diagnosis typical of a pediatric patient and an NTRK fusion

Diseases Treated:

Melanoma

Eligibility:

• Diagnosed with a melanoma tumor that is malignant (cancerous) or that might be cancerous, including:
  • Conventional or “adult-type” melanoma
  • Spitzoid melanoma/atypical Spitz tumor
  • Congenital melanoma
  • Melanoma arising in a giant congenital nevus
  • Melanocytic lesions with indeterminate biological behavior (e.g., pigment-synthesizing melanomas)
• Younger than 19 years of age 

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

Relapsed or refractory solid tumor

Eligibility:

• Participant is at least one month old and younger than 18 years old at the time of signing the informed consent (in Part A, participants < 2 years of age are not included).
• Participant has a histologically confirmed solid tumor
• Participant has a disease that is relapsed or refractory to all potentially curative standard treatment regimens, or has a disease for which there is no standard treatment regimen that is potentially curative.

Diseases Treated:

Advanced melanoma or PD-L1 positive advanced relapsed or refractory solid tumor or lymphoma

Eligibility:

• Between 6 months and 18 years old with diagnosis of MSI-H solid tumor OR
• Between 12 years and 18 years old with diagnosis of melanoma OR
• Between 3 and 18 years old with relapsed or refractory classical Hodgkin lymphoma
• Negative pregnancy test 72 hours prior to medication administration in participants of child-bearing potential
• Appropriate liver and kidney functions

Diseases Treated:

Neuroblastoma

Eligibility:

• Younger than 19 years old
• Newly diagnosed, advanced stage, high-risk neuroblastoma
• Histologic proof of neuroblastoma or positive bone marrow for tumor cells with increased urine catecholamine
• Adequate kidney and liver functions

Diseases Treated:

Neuroblastoma, ganglioneuroblastoma

Eligibility:

• Participant has been enrolled on ANBLB1 (ANBL00B1).
• Participant has newly diagnosed MYCN non-amplified neuroblastoma or MYCN non-amplified ganglioneuroblastoma.
• Participant is less than 12 months (Group A) and less than 18 months (Group B and Group C) of age at diagnosis.

Diseases Treated:

Osteosarcoma

Eligibility:

• At least 12 years old and younger than 25 years old
• Osteosarcoma that has relapsed, progressed or become refractory to conventional therapy

Diseases Treated:

Recurrent, progressive, or refractory high-grade gliomas

Eligibility:

• Participant has recurrent, progressive or refractory non-brainstem high-grade glioma or diffuse intrinsic pontine glioma (DIPG)
• Participant is 1 to 18 years of age
• Participant has bi-measurable disease on MRI 
• Participant has received prior radiation therapy and/or chemotherapy and has not received myelosuppressive anticancer chemotherapy for at least three (3) weeks

Diseases Treated:

Retinoblastoma

Eligibility:

This study is currently open only to patients at St. Jude Children's Research Hospital.

• 7 years old or younger
• Part I
  • Diagnosed with retinoblastoma and has not received any treatment OR
  • Diagnosed with cataracts and has not received any treatment OR
  • Diagnosed with glaucoma and has not received any treatment
• Part II
  • Referred to an eye doctor to check for leukocoria or other eye conditions
• Part III
  • Diagnosed with retinoblastoma and is receiving treatment

Diseases Treated:

Retinoblastoma (non-therapeutic)

Eligibility:

Family history of retinoblastoma with one of the following:

• RB1 mutation OR
• Diagnosis of bilateral retinoblastoma OR
• Diagnosis of unilateral retinoblastoma with RB1 mutation or MYCN amplification

Diseases Treated:

Rhabdomyosarcoma (low-risk, intermediate-risk and high-risk)

Eligibility:

• Newly diagnosed rhabdomyosarcoma
• Younger than 22 years (eligible until 22nd birthday)
• No previous treatment with chemotherapy or radiation therapy

Diseases Treated:

Metastatic sarcomas

Eligibility:

• Participant has histologically or cytologically confirmed diagnosis:
  • Nonrhabdomyosarcoma of soft tissue
  • Ewing sarcoma
  • Osteosarcoma at any site
• Participant must be greater than 3 years of age and < 40 years of age.
• Participant has not had any prior radiotherapy to the treatment site.

Diseases Treated:

Brain Tumor : Recurrent, progressive, or newly diagnosed atypical teratoid rhabdoid brain tumors (ATRT)
Solid Tumor : Progressive extra-CNS malignant rhabdoid solid tumors (MRT)

Eligibility:

• Has one of these types of tumors:
  • Newly diagnosed atypical teratoid rhabdoid tumors (ATRTs) or synchronous extraneural ATRTs
  • ATRTs or malignant rhabdoid tumors (MRTs) that have come back after previous treatment (recurrent disease)
  • ATRTs or MRTs that are growing after previous treatment (progressive disease)
• 21 years of age or younger

Diseases Treated:

Retinoblastoma

Eligibility:

• Newly diagnosed retinoblastoma that has not spread beyond the eye
• Has not received previous treatment with chemotherapy or radiation therapy
• Patients who have been diagnosed with retinoblastoma in one eye who did not receive chemotherapy, but then developed retinoblastoma in the opposite eye

Diseases Treated:

Non-therapeutic

Eligibility:

Primary oncologist:

• Attending physician age 18 years or older who provides medical care to patients in the Solid Tumor Clinic at St. Jude Children’s Research Hospital
• Primary hematology-oncology fellows age 18 or older (audio-recorded disease reevaluation only)

Parents of children with cancer:

• Age 18 or older
• Biological parent, step-parent or primary legal guardian
• Reads and speaks English

Patients:

• Primary oncologist is enrolled in the study.
• 30 years old or younger
• Diagnosed with a solid tumor and/or a brain tumor

Diseases Treated:

Neuroblastoma

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

• All newly diagnosed suspected:
  • neuroblastoma,
  • ganglioneuroblastoma, or 
  • ganglioneuroma (maturing subtype) patients
• Less than or equal to 21 years of age
• Seen at Children's Oncology Group (COG) institutions

Diseases Treated:

Adrenocortical tumors (carcinoma and adenoma)

Eligibility:

• Diagnosis of an adrenocortical tumor (ACT)
• 21 years of age or younger at diagnosis
• Relatives of patients with ACT

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

Diseases Treated:

Influenza

Eligibility:

• At least 3 years old and younger than 17
• Allogeneic hematopoietic stem cell transplant recipient
• At least 3 and not more than 35 months after transplant
• Platelet count of at least 30,000

Eligibility:

• Child is greater than or equal to twelve (12) months and less than twenty-four (24) months.
• Child has adequate blood, liver and kidney function.
• Child has no history of lung disease, asthma, and hospitalization for respiratory illness, immunodeficiency, sickle cell disease, or any other serious underlying condition.