Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma

Conditions

• Posterior Fossa Ependymal Tumor

Eligibility Criteria

Sex

ALL

Age

1 Year - 39 Years

Healthy Volunteers

Not accepted

Interventions

• Metformin — DRUG
  Given orally (PO)
• Planned Surgical Resection — PROCEDURE
  Undergo planned surgery as part of regular care
• Magnetic Resonance Imaging (MRI) — PROCEDURE
  Undergo MRI imaging
• Specimen Collection — PROCEDURE
  Tumor Tissue, blood, and cerebral spinal fluid (CSF) may be collected for correlative analysis.
• MR spectroscopy (MRS) — PROCEDURE
  Undergo MRS imaging

Study Details

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.

Key Dates

Start date

May 5, 2026

Status verified

Jun 2026

Primary completion

Apr 30, 2028

Completion

Mar 31, 2030

Study Design

Enrollment

30 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Target Validation (TV) Phase (Metformin)
  Participants will each receive daily oral metformin with a weekly schedule starting at 250 mg/m\^2 twice a day (BID) for the first week, then increasing to 500 mg/m\^2 BID for week 2, 1000 mg/m\^2 BID for week 3, and then 1666 mg/m\^2 BID for week 4-6 prior to planned surgery. Participants with no measurable disease following surgical intervention may either go off treatment or continue metformin at the physician's discretion. Participants have the option of continuing on the efficacy phase of the study without undergoing planned surgery no demonstrated progressive disease on pre-surgery MRI AND if the family opts to continue treatment with metformin without undergoing surgical resection of the tumor. Participants with measurable tumor by RAPNO criteria following surgery may continue onto efficacy phase and receive maintenance metformin until disease progression or till there are unacceptable adverse event(s).
• Experimental: Efficacy Phase (Metformin)
  Participants with recurrent or progressive PFA ependymoma that have failed upfront surgery and radiation will receive daily oral metformin until disease progression or till there are unacceptable adverse event(s). Disease response will be assessed by MRI imaging using RAPNO criteria. Participants already enrolled on the TV phase may enroll in this phase if they have measurable disease post-surgery. Treatment may continue until disease progression or until there are unacceptable adverse event(s).

Primary Outcome Measure

Proportion of participants with changes in biomarkers between the pre-and post-metformin treated samples (Target Validation Phase) [ Time Frame: From initiation of study treatment until surgical resection of tumor, approximated 6 weeks ]

Central Contacts

• PNOC Operations Office
  415-502-1600
  [email protected]

Locations (1)

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