RecruitingPhase 2

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

United States118 participantsStarted 2021-03-29

Plain-language 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.

Who can participate

Age range21 Years

SexALL

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Inclusion criteria

✓. Age: 0-21 years of age at diagnosis
✓. Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.
✓. Pre-enrollment tumor survey:
✓. Disease Status: Subjects must have no evidence of disease, or stable\* residual nonbulky\*\* disease.
✓. Timing from prior therapy:
✓. 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.
✓. 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.
✓. Patients must have adequate organ functions at the time of registration:

Exclusion criteria

✕. BSA of \<0.25 m2
✕. Metastatic disease outside of CNS
✕. Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
✕. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
✕. 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.
✕. 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.
✕. 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.

What they're measuring

Number of participants with event free survival (EFS) during study

Timeframe: 2 years plus 5 years follow up

Trial details

NCT IDNCT04696029

SponsorGiselle Sholler

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-03

Contact for this trial

BCC Enroll

7175310003 BCCEnroll@pennstatehealth.psu.edu

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