Phase I / II Clinical Trials

Diseases Treated:

Newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

Eligibility:

• Participant is > 36 month and < 21 years of age at the time of study enrollment.
• Participant has newly diagnosed DIPG.
• Participant has a brainstem tumor that was biopsied and proven to be one of the following: anaplastic astrocytoma, glioblastoma, gliosarcoma, or anaplastic mixed glioma.
• Participant has not received any prior chemotherapy, radiation therapy, immunotherapy or bone marrow transplant for the treatment of DIPG. Prior dexamethasone and/or surgery are allowed.

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:

Solid Tumor, CNS Tumor, Lymphoma

Eligibility:

• 12 months to 21 years old
• Able to swallow whole tablets
• Diagnosis of recurrent or refractory solid tumor, including CNS tumor and lymphoma

Diseases Treated:

Solid Tumor, CNS Tumor

Eligibility:

• 12 months to 21 years old
• Diagnosis of recurrent or refractory solid tumor, including CNS tumor

Diseases Treated:

Solid tumors, giomas, hystiocytosis

Eligibility:

• Participant is greater or equal to 12 months and less than 18 years at the time of signing the informed consent form.
• Participant has BRAF V600 mutation positive tumor as confirmed in a CLIA-approved laboratory or equivalent
• Participant has recurrent disease, refractory disease, or progressive disease after having received at least one standard therapy for their disease, OR participant has metastatic (or surgically unresectable) melanoma, and is being enrolled for first-line treatment. Melanoma participants with CNS involvement may be enrolled.

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:

Newly diagnosed localize primary NGGCT or newly diagnosed localized germinoma

Eligibility:

• Participant is > 3 years and < 21 years at the time of enrollment.
• Participant has newly diagnosed localized primary NGGCT or newly diagnosed localized germinoma (has not spread to other parts of the brain or spine).
• Participant must have adequate bone marrow function.
• Participant must have normal renal and liver function for age.
• NO prior therapy except surgery and/or steroids.

Diseases Treated:

Human papillomavirus

Eligibility:

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

Questionnaire phase

• Cancer survivor (9 to 26 years of age at study participation)
• English or Spanish-speaking

Vaccine Phase

• Survey response indicated no prior history of HPV vaccination
• Agrees to return to participating institution for 3 HPV vaccine injections
• Allergy to any component of the HPV vaccine including yeast and aluminum will not be eligible for the 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:

• Rhabdoid tumor
• Synovial sarcoma
• INI 1-negative tumor

Eligibility:

• Between 6 months and 21 years old
• Relapsed or refractory rhabdoid tumors, INI1-negative tumors or synovial sarcoma

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:

Respiratory Syncytial Virus (RSV)

Eligibility:

• 6 to 24 months old
• In good health
• Never been diagnosed with Respiratory Syncytial Virus (RSV)
• Growing at normal rate for age
• Received routine immunizations, appropriate for age

Diseases Treated:

Refractory or progressive malignancies

Eligibility:

• Age > 12 months and < 18 years.
• Participant has been diagnosed with a locally advanced or metastatic malignancy that has progressed despite standard therapy, or which no effective standard therapy exists.
• Specimen is being submitted for molecular pre-screening to test for genetic alteration of ALK and the tumor must carry a genetic alteration of the ALK gene.
• Participant has adequate liver, kidney, cardiac, and bone marrow function.

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

• 1 to 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
• ≥ 1 month old with diagnosis of infantile/congenital fibrosarcoma or congenital mesoblastic nephroma with documented NTRK fusion that has progressed or was nonresponsive to therapy and for which there is no standard or available curative therapy

Phase 2

• ≥ 1 month old at C1D1 with locally advanced or metastatic infantile fibrosarcoma OR
• ≥ 1 month old through 21 years old at C1D1 with locally advanced or metastatic solid tumor or primary CNS tumor that has 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
• Weight:
  • Males: more than 55 kilograms (about 121 pounds)
  • Females: more than 50 kilograms (about 110 pounds)
• Lifetime cumulative anthracycline dose: ≥ 300 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:

Sickle cell disease

Eligibility:

• 12 to 17 years old
• Diagnosis of sickle cell disease
• Able to swallow capsules

 

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:

Unresectable or recurrent craniopharyngioma

Eligibility:

• Participant has a histologically verified diagnosis of craniopharyngioma.
• Participant has recovered from acute toxicities of all prior therapy before entering this study.
• Participant has not received prior interferon, either systemic or into the tumor cyst.
• Participant does not have evidence of metastatic tumor.

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:

Refractory and recurrent solid tumors

Eligibility:

• Participant is between 1-25 years old e at the time of enrollment (Note:  eligibility up to age 50 years for other participating sites).
• Participant has had histologic verification of an extracranial germ cell tumor that is not a pure teratoma (mature or immature), pure germinoma, or pure seminoma.
• Participant has relapsed or refractory disease following at least two prior platinum-containing chemotherapy regimens.
• Patients must have measurable disease, documented according to RECIST criteria, or evaluable disease with a standard tumor marker (AFP and/or HCG) greater than 10 times the upper limit of normal.
• Other requirements for this study.

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:

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