Phase I / II Clinical Trials

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:

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:

 Low-grade glioma

Eligibility:

• At least 6 years old and younger than 22
• Diagnosis of low-grade glioma

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:

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:

Brain Tumors

Eligibility:

• Between 6 and 21 years old
• Diagnosis of localized low-grade glioma, craniopharyngioma, 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

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:

• 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:

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 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

Diseases Treated:

Medulloblastoma

Eligibility:

Eligibility Overview

• Recurrent, refractory or progressive medulloblastoma
• At least 1 year old and younger than 25 years old

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