Phase I / II Clinical Trials

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:

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:

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:

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:

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:

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:

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:

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