RECRUITING

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

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Quick Facts

Study Start:2021-03-29

Study Completion:2029-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04696029

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 21 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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. * Metastatic non-MYC amplified Group 3 * Metastatic Group 4 * Metastatic non-WNT/non-SHH (Must be non-MYC amplified) * Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH * MYC amplified Group 3 * Non-WNT, non-SHH infant (\< 3 yrs) 3. Pre-enrollment tumor survey: * 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. 5. Timing from prior therapy: 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).	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.

Inclusion Criteria

Exclusion 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.
* Metastatic non-MYC amplified Group 3
* Metastatic Group 4
* Metastatic non-WNT/non-SHH (Must be non-MYC amplified)
* Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH
* MYC amplified Group 3
* Non-WNT, non-SHH infant (\< 3 yrs)
3. Pre-enrollment tumor survey:
* 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.
5. Timing from prior therapy:
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).

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.

Contacts and Locations

Study Contact

BCC Enroll

CONTACT

7175310003

BCCEnroll@pennstatehealth.psu.edu

Principal Investigator

Michael A Huang, MD

STUDY_CHAIR

Beat Childhood Cancer at Atrium Health

Study Locations (Sites)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202

United States

UCSF Benioff Children's Hospital Oakland-

Oakland, California, 94609

United States

Rady Children's Hospital

San Diego, California, 92123

United States

Connecticut Children's Hospital

Hartford, Connecticut, 06106

United States

Arnold Palmer Hospital for Children

Orlando, Florida, 32806

United States

St. Joseph's Children's Hospital

Tampa, Florida, 33607

United States

Kentucky Children's Hospital

Lexington, Kentucky, 40502

United States

University of Louisville/Norton's Children's

Louisville, Kentucky, 40202

United States

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108

United States

Cardinal Glennon Children's Medical Center

Saint Louis, Missouri, 63104

United States

Hackensack University Medical Center

Hackensack, New Jersey, 07601

United States

Levine Children's Hospital

Charlotte, North Carolina, 28204

United States

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

Hershey, Pennsylvania, 17033

United States

Medical University of South Carolina

Charleston, South Carolina, 29425

United States

Dell Children's Blood and Cancer Center

Austin, Texas, 78723

United States

Collaborators and Investigators

Sponsor: Giselle Sholler

Michael A Huang, MD, STUDY_CHAIR, Beat Childhood Cancer at Atrium Health

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-03-29

Study Completion Date2029-03

Study Record Updates

Study Start Date2021-03-29

Study Completion Date2029-03

Terms related to this study

Additional Relevant MeSH Terms

Medulloblastoma