NCT04696029

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 118 (estimated)
• Sponsor: Giselle Sholler · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

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.

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)

Conditions

• Medulloblastoma

Interventions

Type	Name	Description
DRUG	Difluoromethylornithine	DFMO (difluoromethylornithine is an inhibitor of ornithine decarboxylase (ODC) designated chemically as 2-(difluoromethyl)-DL-ornithine monohydrochloride monohydrate. The dosage form to be used in this study is provided as a convex tablet containing 192 mg eflornithine (equivalent to 250 mg of eflornithine HCl, monohydrate). The tablets are packaged and sealed in opaque white HDPE bottles, and each bottle contains 100 tablets. The DMFO tablets are supplied by USWorldMeds (USWM). The tablets are to be stored at room temperature (20-250C).

Timeline

• Start date: 2021-03-29
• Primary completion: 2028-03-01
• Completion: 2029-03-01
• First posted: 2021-01-06
• Last updated: 2026-03-02

Locations

23 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study