Phase I / II Clinical Trials

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 BRAF^V600 mutation

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:

Solid Tumors 
Leukemias

Eligibility:

• 21 years old or younger (at St. Jude)
• Newly diagnosed infantile fibrosarcoma  or solid tumor with NTRK1, NTRK2 or NTRK3 fusion OR
• Relapsed/refractory acute leukemia with NTRK1, NTRK2 or NTRK3 fusion

Diseases Treated:

Sickle Cell Disease

Eligibility:

• 18 to 35 years old (Ages 18 to 25 at St. Jude)
• Diagnosis of severe sickle cell disease (SCD)

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.

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:

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:

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:

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

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:

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

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:

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:

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

Diseases Treated:

Acute Myeloid Leukemia (AML)

Eligibility:

• Younger than 21 years of age
• Diagnosed with recurrent or refractory acute myeloid leukemia (AML)
• Weigh more than 17kg 
• Fully recovered from the acute toxic effects of all prior anti-cancer therapy

Diseases Treated:

Diffuse Intrinsic Pontine Glioma, Glioma, CNS Tumor, Solid Tumor

Eligibility:

• Diagnosed with one of the following:
  • Recurrent or refractory solid tumor or central nervous system (CNS) tumor
  • Diffuse Intrinsic Pontine Glioma  (DIPG)
  • Newly diagnosed or recurrent high-grade glioma 
• Younger than 18 years old (Phase I) or between 3 and 25 years old (Efficacy Phase)

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.

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:

Solid tumors, brain tumors, CNS tumors

Eligibility:

• Diagnosed with one of the following:
• Relapsed or refractory extracranial solid tumor
• Primary brain tumor with NTRK1/2/3, ROS1 or ALK gene fusion
• Extracranial solid tumor with NTRK1/2/3, ROS1 or ALK gene fusion
• Younger than 22 years old
• Not pregnant

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