Diabetes drug could fight rare brain cancer in kids
NCT ID NCT07226453
First seen Jun 24, 2026 · Last updated Aug 25, 2026 · Updated 9 times
Summary
This study tests whether metformin, a common diabetes medicine, can help children and young adults with a rare brain tumor called PFA ependymoma that has come back. About 30 participants will take metformin and have surgery and imaging to see if the drug changes certain proteins in the tumor and slows its growth. The goal is to find a new way to control this hard-to-treat cancer.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Metformin (Glucophage)
- What this could lead to
- If successful, this could point toward a new treatment option for a rare brain tumor that currently has few options after standard therapy.
- What could go wrong
- This is a small, early-phase trial with only 30 participants. Metformin is not a standard cancer drug, and it may not slow tumor growth or improve outcomes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2026
- Expected to finish
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Mar 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 year to 39 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Participants must have recurrent or progressive posterior fossa A (PFA) ependymoma following surgery AND radiation treatment (RT). 2. Participants must have a diagnosis of PFA ependymoma. Any number of previous recurrences are permissible provided the participant meets other enrollment criteria. 3. 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. Target Validation (TV) Phase: o Participants are candidates to undergo elective surgery for removal of all or a portion of their recurrent/progressive tumor. Efficacy Phase: * Participant must have measurable disease; this will be defined as lesions that can be accurately measured in two dimensions (longest diameter to be recorded) The size threshold is met if both in-plane diameters are ≥10 mm or both in-plane diameters are at least two times the MRI slice thickness, plus the interslice gap with a minimum size of no less than double the slice thickness on MRI. * Participants with an isolated local progression of the tumor following RT (or stereotactic radiosurgery, SRS) must be \> 6 months from completion of RT to the lesion to rule out pseudo progression or must have tissue confirmation of progression prior to enrollment. * Previously irradiated lesions are considered non-measurable except in cases of documented progression of the lesion since the completion of radiation therapy as outlined above. 4. 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. 5. Age: 1 -39 years at the time of enrollment. 6. 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. 7. Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration. 8. Organ Function Requirements 1. Peripheral absolute neutrophil count (ANC) \>= 1000/mm\^3. 2. Platelet count \>=100,000/mm\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment). 3. Serum creatinine \< 1.5 Upper Limit normal (ULN) based on age and gender 4. Total bilirubin \<= 1.5 x ULN for age; in presence of Gilbert's syndrome, total bilirubin \< 3 x ULN or direct bilirubin \< 1.5 x ULN 5. alanine aminotransferase (ALT) \<= 3 x ULN. 6. aspartate aminotransferase (AST) \<=3 x ULN. 9. Participants with seizure disorder may be enrolled if well controlled and are on stable dose of anti-seizure medication for \> 72 hours prior to enrollment. Participants who have neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration. 10. Participants must enroll on PNOC COMP if PNOC COMP is open to accrual at the enrolling institution. 11. A legal parent/guardian or participant must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate. Exclusion Criteria: 1. Participants with a history of diabetes mellitus or those meeting criteria for pre-diabetes are excluded. Pre-diabetes is defined per American Diabetes Association criteria as any of the following: fasting plasma glucose (FPG) 100-125 mg/dL (5.6-6.9 mmol/L); 2-hour plasma glucose (OGTT) 140-199 mg/dL (7.8-11.0 mmol/L); or hemoglobin A1c (HbA1c) 5.7%-6.4% (39-47 mmol/mol). 2. Participants without any measurable disease. 3. 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. 4. 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. 5. 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 6. Participants who are receiving any other investigational agents. 7. Concurrent radiation therapy in any form is not permitted while on the study drug 8. History of allergic reactions attributed to compounds of similar chemical or biologic composition to metformin. 9. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection. 10. Women of childbearing potential must not be pregnant or breast-feeding. 11. Human immunodeficiency virus- (HIV) positive participants will be ineligible if HIV therapy regimen has not been stable for at least 4 weeks or there is intent to change the regimen within 8 weeks following enrollment, or if they are severely immunocompromised.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
10 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Children's National Medical Center
NOT_YET_RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Duke University
RECRUITINGDurham, North Carolina, 27710, United States
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Hackensack Meridian Health
NOT_YET_RECRUITINGHackensack, New Jersey, 07601, United States
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Johns Hopkins University
RECRUITINGBaltimore, Maryland, 21287, United States
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St. Jude Children's Research Hospital
NOT_YET_RECRUITINGMemphis, Tennessee, 38105, United States
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
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University of California, San Francisco
RECRUITINGSan Francisco, California, 94143, United States
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University of Michigan
NOT_YET_RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Utah
NOT_YET_RECRUITINGSalt Lake City, Utah, 84113, United States
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Washington University
RECRUITINGSt Louis, Missouri, 63110, United States