Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependy… (NCT07226453) | Clinical Trial Compass
RecruitingPhase 2
Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma
United States30 participantsStarted 2026-05-05
Plain-language summary
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.
Who can participate
Age range1 Year – 39 Years
SexALL
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Inclusion criteria
✓. Participants must have recurrent or progressive posterior fossa A (PFA) ependymoma following surgery AND radiation treatment (RT).
✓. Participants must have a diagnosis of PFA ependymoma either at initial diagnosis or at recurrence. Any number of previous recurrences are permissible provided the participant meets other enrollment criteria.
✓. Participants must have adequate tumor tissue available from initial diagnosis or from pre- trial enrollment. Formalin-fixed paraffin-embedded (FFPE) material (1 full block) should be provided. If FFPE material is not available, 10 unstained slides with an accompanying hematoxylin and eosin (H\&E) report should be provided.
✓. Prior Therapy: Participants must not be receiving metformin for other medical indications or previous exposure to metformin following their diagnosis of PFA ependymoma. However, participants treated on the TV phase, but did not continue onto maintenance therapy will be allowed to enroll on the efficacy phase with future recurrences or progression of their disease.
✓. Age: 1 -39 years at the time of enrollment.
✓. Performance Score: Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>=50 for participants \<=16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
✓. Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration.
✓. Organ Function Requirements
Exclusion criteria
What they're measuring
1
Proportion of participants with changes in biomarkers between the pre-and post-metformin treated samples (Target Validation Phase)
Timeframe: From initiation of study treatment until surgical resection of tumor, approximated 6 weeks
2
Disease Stabilization Rate (Efficacy Phase)
Timeframe: From initiation of study treatment until discontinuation of treatment, up to 2 years
. Participants with a history of diabetes mellitus or those found to have pre-diabetes on HbA1C screening test are excluded from the study.
✕. Participants without any measurable disease but with only isolated leptomeningeal disease progression.
✕. Participants who have had chemotherapy or have received radiotherapy to the non-target lesion within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
✕. Participants must be at least 7 days since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events that can occur beyond 7 days after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur. Such participants should also be discussed with study chairs.
✕. Participants with rapidly progressive symptoms that require urgent surgery that in the investigators assessment cannot be safely deferred for 6 weeks are excluded from target validation phase of the study
✕. Participants who are receiving any other investigational agents.
✕. History of allergic reactions attributed to compounds of similar chemical or biologic composition to metformin.
✕. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.