Phase I / II Clinical Trials

 

St. Jude Clinical Trials

ED0157: Phase 1 Pediatric PK/PD Study

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.
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FIX-GT: Factor IX Gene Therapy Study for Patients with Hemophilia B

Diseases Treated:

Hemophilia B

Eligibility:

  • Male
  • At least 18 years old
  • Diagnosis of hemophilia B
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FLOPET: PET Scan Studies to Analyze Neuroblastoma and Pheochromocytoma Tumors

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

 

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HALGG: Hippocampal-Avoidance Using Proton Therapy in Children with Brain Tumors

Diseases Treated:

 Low-grade glioma

Eligibility:

  • At least 6 years old and younger than 22
  • Diagnosis of low-grade glioma
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INOMRD: Inotuzumab Ozogamicin for Children with High-Risk B-Cell ALL

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
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iRET: Intravitreal Chemotherapy for Children with Retinoblastoma

Diseases Treated:

Retinoblastoma

Eligibility:

  • Age 17 or younger
  • Diagnosis of retinoblastoma that did not go away with treatment or came back after treatment
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LTE1621: Pharmacologic Reversal of Ventricular Remodeling in Childhood Cancer Survivors at Risk for Heart Failure (PREVENT-HF): A Phase 2B Randomized Placebo-Controlled (Carvedilol) Trial

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
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MEMCRT: Memantine to Prevent Thinking Problems in Children Receiving Radiation for Certain Brain Tumors

Diseases Treated:

Brain Tumors

Eligibility:

  • Between 6 and 21 years old
  • Diagnosis of localized low-grade gliomacraniopharyngioma, ependymoma, meningioma 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
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PMVOCVR: Virtual Reality Therapy for Vaso-Occlusive Crisis in Patients with Sickle Cell Disease

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
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REF2HCT: Haploidentical Bone Marrow Transplant for Leukemia and Lymphoma

Diseases Treated:

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
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RERTEP: Surgery and Second-Course Radiation Therapy in Treating Younger Patients With Recurrent Ependymoma

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
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RT3CR: Proton Therapy for Pediatric Craniopharyngioma

Diseases Treated:

Craniopharyngioma

Eligibility:

  • 21 years old or younger
  • Diagnosis of craniopharyngioma
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SELHEM: Selinexor With Fludarabine and Cytarabine for Treatment of Refractory or Relapsed Leukemia or Myelodysplastic Syndrome

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
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SENDAI: A Phase I Study of Unmodified Live Intranasal Sendai Virus Vaccine in Children and Toddlers: Assessment of Safety and Immunogenicity

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.
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SJATRT: Phase II Study of Alisertib Therapy for Rhabdoid Tumors

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
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SJDAWN: Molecular-Based Therapy for Aggressive Brain and Spinal Cord Tumors in Children and Young Adults

Diseases Treated:

Brain tumor, medulloblastoma, ependymoma, high-grade glioma (HGG), CNS embryonal tumors, and atypical 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
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SJELIOT: Phase I Study of a CHK1/2 Inhibitor Therapy in Combination with Chemotherapy for Children and Adolescents with Refractory or Recurrent Medulloblastoma Brain Tumors

Diseases Treated:

Medulloblastoma

Eligibility:

Eligibility Overview

  • Recurrent, refractory or progressive medulloblastoma
  • At least 1 year old and younger than 25 years old
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SJMB12: A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma/PNET

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
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SJPI3K: Phase I Study of GDC-0084 in Young Patients with Newly Diagnosed DIPG or other Gliomas after Radiation Therapy

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
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SJRET6: Combination Chemotherapy in Treating Patients with Newly Diagnosed, Previously Untreated Intraocular Retinoblastoma

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
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TINI: Total Therapy for Infants With 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
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Collaborative Clinical Trials

ACNS1721: A Study of the Drug Veliparib with Radiation Therapy in Patients with Newly Diagnosed High-Grade Glioma

Diseases Treated:

High-grade glioma

Eligibility:

  • 3 to 25 years old
  • Newly diagnosed high-grade-glioma[SJ1] , such as anaplastic astrocytoma or glioblastoma
  • Negative test for H3 K27M mutation
  • Negative test for BRAFV600 mutation
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ADV1312: WEE1 Inhibitor MK-1775 and Irinotecan Hydrochloride in Treating Younger Patients With Relapsed or Refractory Solid Tumors

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.
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ADV1414: Phase I Study of Selinexor to Treat Patients with Recurrent and Refractory Pediatric Solid Tumors

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
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DRBHGG: Combination Chemotherapy for Low Grade Glioma (LGG) and Relapsed or Refractory High Grade Glioma (HGG) Brain Tumors

Diseases Treated:

High grade glioma (HGG), low grade glioma (LGG)

Eligibility:

  • At least 12 months old and younger than 18 years of age
  • High grade glioma with BRAF V600 mutation that has relapsed, progressed or failed to respond to therapy OR
  • Newly diagnosed Low grade glioma with BRAF V600 mutation that is not treatable with surgery and no previous treatment with chemotherapy, radiotherapy or targeted agents.
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ELVIS: Two Part Study to Study Pharmacokinetics, Safety, and Antiviral Activity of Elvitegravir (EVG) Administered With a PI/r Background Regimen for ARV Treatment-Experienced Pediatric Subjects

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.
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FIX-GT: Factor IX Gene Therapy Study for Patients with Hemophilia B

Diseases Treated:

Hemophilia B

Eligibility:

  • Male
  • At least 18 years old
  • Diagnosis of hemophilia B
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HODLP: First International Inter-Group Study for Nodular Lymphocyte Predominant Hodgkin Lymphoma in Children and Adolescents

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

 

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LDTAM: Low-Dose Tamoxifen Citrate in Reducing Breast Cancer Risk in Radiation-Induced Cancer Survivors

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.
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LOXO-TRK: Testing Larotrectinib (LOXO-101) in Children with Advanced Solid Tumors or Brain Tumors

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
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LOXORET: Phase I/II Study of LOXO-292 in Patients with Advanced RET-Altered Solid Tumors or Brain Tumors

Diseases Treated:

Solid tumors, Brain tumors

Eligibility:

  • 6 months to 21 years old
  • Solid tumor or brain tumor that has progressed or spread
  • Failed standard treatment
  • RET gene alteration
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LTE1621: Pharmacologic Reversal of Ventricular Remodeling in Childhood Cancer Survivors at Risk for Heart Failure (PREVENT-HF): A Phase 2B Randomized Placebo-Controlled (Carvedilol) Trial

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
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MEKPEM: A Phase I/II trial of MK-3475 (pembrolizumab) in children’s solid tumors and lymphomas

Diseases Treated:

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

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
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P1093: Safety of and Immune Response to Dolutegravir in HIV-1 Infected Infants, Children, and Adolescents

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.)
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PBTC29: Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

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.
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PBTC47: A Clinical Trial of the Drug, Panobinostat, in Children with Brain Tumors

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
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PBTC49: Study of Savolitinib in Recurrent, Progressive or Refractory Medulloblastoma, HGG or DIPG

Diseases Treated:

Medulloblastoma

High-grade glioma (HGG)

Diffuse intrinsic pontine glioma (DIPG)

Eligibility:

  • Recurrent, refractory or progressive medulloblastoma, high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG)
  • Evidence of genetic activation of the MET pathway (expansion co-hort)
  • At least 5 years old and 21 years old or younger
View Trial

SBRT1: Stereotactic Radiation Therapy for Pediatric Sarcomas

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.
View Trial

SENDAI: A Phase I Study of Unmodified Live Intranasal Sendai Virus Vaccine in Children and Toddlers: Assessment of Safety and Immunogenicity

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.
View Trial

TINI: Total Therapy for Infants With 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
View Trial