Actively Recruiting
Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma
Led by University of California, San Francisco · Updated on 2026-05-07
30
Participants Needed
1
Research Sites
203 weeks
Total Duration
On this page
Sponsors
U
University of California, San Francisco
Lead Sponsor
T
The Lilabean Foundation, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a multi-site study of the pharmacodynamic effects and efficacy of metformin in children and young adults with recurrent or progressive Posterior Fossa Group A (PFA) ependymoma.
CONDITIONS
Official Title
Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must have recurrent or progressive posterior fossa A (PFA) ependymoma following surgery and radiation treatment.
- Participants must have a diagnosis of PFA ependymoma either at initial diagnosis or at recurrence; any number of previous recurrences allowed.
- Adequate tumor tissue must be available from initial diagnosis or before trial enrollment, either as formalin-fixed paraffin-embedded material or unstained slides with report.
- TV Phase: Participants must be candidates for elective surgery to remove all or part of their recurrent/progressive tumor.
- Efficacy Phase: Participants must have measurable disease defined by specific MRI size criteria.
- Participants with isolated local tumor progression after radiation must be more than 6 months post-radiation or have tissue confirmation of progression.
- Previously irradiated lesions are non-measurable except if progression since radiation is documented.
- Participants must not be receiving metformin for other medical indications or have previous metformin exposure after PFA diagnosis, except those from TV phase enrolling later.
- Age between 1 and 39 years at enrollment.
- Karnofsky score ≥ 50 for those over 16 years; Lansky score ≥ 50 for those 16 years or younger; wheelchair-bound but ambulatory participants are eligible.
- Stable or decreasing dose of dexamethasone for at least 1 week prior to registration.
- Adequate organ function including specific blood counts, kidney and liver function measures.
- Participants with well-controlled seizure disorder on stable medication dose for over 72 hours.
- Neurological deficits must be stable for at least 1 week before registration.
- Must enroll on PNOC COMP if open at enrolling institution.
- Legal guardian or participant able to understand and sign informed consent and assent as appropriate.
You will not qualify if you...
- History of diabetes mellitus or pre-diabetes based on HbA1C screening.
- Participants with only isolated leptomeningeal disease progression without measurable disease.
- Chemotherapy or radiotherapy to non-target lesion within 3 weeks (6 weeks for specific agents) or unresolved adverse events from earlier treatments.
- Less than 7 days since completion of therapy with biologic or small molecule agents, or within time adverse events are known to occur.
- Rapidly progressive symptoms requiring urgent surgery that cannot be deferred safely for 6 weeks (for TV phase).
- Receiving any other investigational agents.
- History of allergic reactions to compounds similar to metformin.
- Uncontrolled active infection or other serious illness.
- Pregnant or breastfeeding women of childbearing potential.
- HIV-positive participants with unstable therapy, planned therapy changes, or severe immunocompromise.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
Research Team
P
PNOC Operations Office
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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