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

ConditionMedulloblastoma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 21
SponsorGiselle Sholler

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Metastatic non-MYC amplified Group 3

Metastatic Group 4

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Difluoromethylornithine

    Drug

    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).

Treatment groups

118 Participants
are divided into 1 treatment group
Group A: Difluoromethylornithine (DFMO)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

o To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in preventing relapse in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon the 2-year progression-free survival rate (PFS) compared to relevant historical controls.

Time frame
2 years plus 5 years follow up

Secondary outcomes

1

Length of time that participants experience Overall Survival (OS)

o To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon overall survival

Time frame
7 years
2

Determine the Overall Response Rate (ORR) of Participants using Modified RANO Criteria

To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon Response Rate for patients with non-bulky residual disease present.

Time frame
2 years
3

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

To develop a complete safety and tolerability profile of difluoromethylornithine (DFMO) in pediatric and young adult subjects with medulloblastoma.

Time frame
2 years plus 30 days
4

Determine amount of DFMO in the CSF at 3 hours post dose

o To measure CSF penetration after DFMO administration in pediatric subjects with medulloblastoma

Time frame
2 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Giselle Sholler

Lead sponsor

Milton S. Hershey Medical Center

Sponsor institution

Source ClinicalTrials.gov
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