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NCT04696029 | RECRUITING | Medulloblastoma

DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
Sponsor:

Gielle Sholler

Information provided by (Responsible Party):

Gielle Sholler

Brief Summary:

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma.

Condition or disease

Medulloblastoma

Intervention/treatment

Difluoromethylornithine

Phase

PHASE2

Detailed Description:

In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 2500 mg/m2 BID on each day of study. Subjects will be evaluated in 3 Cohorts: Cohort 1: Molecular High Risk Medulloblastoma Cohort 2: Molecular Very High Risk Medulloblastoma Cohort 3: Relapsed/Refractory Medulloblastoma A total of 118 subjects across all cohorts will be enrolled to ensure that there will be 107 evaluable subjects (32-39 per cohort)

Study Type : INTERVENTIONAL
Estimated Enrollment : 118 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma
Actual Study Start Date : 2021-03-29
Estimated Primary Completion Date : 2028-03
Estimated Study Completion Date : 2029-03

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: to 21 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • 1. Age: 0-21 years of age at diagnosis
  • 2. Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.
  • Cohort 1- Molecular High Risk:
  • * Metastatic non-MYC amplified Group 3
  • * Metastatic Group 4
  • * Metastatic non-WNT/non-SHH (Must be non-MYC amplified)
  • Cohort 2- Molecular Very High Risk
  • * Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH
  • * MYC amplified Group 3
  • * Non-WNT, non-SHH infant (\< 3 yrs)
  • Cohort 3: Relapsed/Refractory Medulloblastoma
  • 3. Pre-enrollment tumor survey
    • Prior to enrollment on this study, a determination of mandatory disease staging must be performed:
    • * Tumor imaging studies including: Brain and spine MRI
    • * Lumbar Puncture only if previously positive
    • * Bone Marrow aspiration/biopsy only if previously positive
    • * This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.
    • 4. Disease Status: Subjects must have no evidence of disease, or stable\* residual nonbulky\*\* disease.
    • \*Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart
    • \*\*Non-bulky disease defined as maximal cross-sectional area \< 3cm\^2 at enrollment. Patients with leptomeningeal disease are allowed to participate on study.
    • 5. Timing from prior therapy
      • Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell transplantation (SCT) are eligible if more than 45 days have elapsed since date of last SCT.
      • 6. Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.
      • 7. All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
      • 8. Patients must have adequate organ functions at the time of registration
        • * Hematological: Hematological recovery as defined by ANC ≥750/μL, platelets ≥30 (non-transfused x 7 days)
        • * Liver: Adequate liver function as defined by AST and ALT \<10x upper limit of normal
        • * Renal: Adequate renal function defined as (perform one of the following): Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or a serum creatinine based on age/gender
        • 9. Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
        • 10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).
        Exclusion Criteria
        • 1. BSA of \<0.25 m2
        • 2. Metastatic disease outside of CNS
        • 3. Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
        • 4. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
        • 5. Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
        • 6. Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
        • 7. Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

Location Details

NCT04696029

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, Arkansa

Arkansas Children's Hospital

Little Rock, Arkansa, United States, 72202

RECRUITING

United States, California

UCSF Benioff Children's Hospital Oakland-

Oakland, California, United States, 94609

RECRUITING

United States, California

Rady Children's Hospital

San Diego, California, United States, 92123

NOT YET RECRUITING

United States, Connecticut

Connecticut Children's Hospital

Hartford, Connecticut, United States, 06106

RECRUITING

United States, Florida

Arnold Palmer Hospital for Children

Orlando, Florida, United States, 32806

RECRUITING

United States, Florida

St. Joseph's Children's Hospital

Tampa, Florida, United States, 33607

RECRUITING

United States, Kentucky

Kentucky Children's Hospital

Lexington, Kentucky, United States, 40502

RECRUITING

United States, Kentucky

University of Louisville/Norton's Children's

Louisville, Kentucky, United States, 40202

RECRUITING

United States, Missouri

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, United States, 64108

NOT YET RECRUITING

United States, Missouri

Cardinal Glennon Children's Medical Center

Saint Louis, Missouri, United States, 63104

RECRUITING

United States, New Jersey

Hackensack University Medical Center

Hackensack, New Jersey, United States, 07601

RECRUITING

United States, North Carolina

Levine Children's Hospital

Charlotte, North Carolina, United States, 28204

RECRUITING

United States, Pennsylvania

Penn State Milton S. Hershey Medical Center and Children's Hospital

Hershey, Pennsylvania, United States, 17033

RECRUITING

United States, South Carolina

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

RECRUITING

United States, Texas

Dell Children's Blood and Cancer Center

Austin, Texas, United States, 78723