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# Metformin Tested in Children With Recurrent PFA Ependymoma — Phase 2 Trial Recruiting at Six US Centers
- URL: https://www.sanarelab.science/metformin-pfa-ependymoma-trial-children-recruiting-2026/
- Published: 2026-10-15T08:18:25.000Z
- Updated: 2026-10-15T08:18:25.000Z
- Description: NCT07226453 (UCSF, six US centers) is recruiting patients aged 1–39 with recurrent or progressive PFA ependymoma to test whether metformin changes the tumor's epigenetic markers (EZHIP/H3K27me3) and stabilizes disease. No results yet.
- Author: Sanare Lab
- Tags: #short-read, News, Research, Repurposed Drugs

Key Takeaway

A US trial is recruiting children and young adults (ages 1–39) with recurrent PFA ependymoma — an aggressive brain tumor — to test whether the common diabetes drug metformin can switch the tumor's epigenetic settings back toward normal. No results yet; this is a trial announcement.

PFA ependymoma is a childhood brain tumor driven not by DNA mutations but by epigenetics — chemical settings that control which genes are active. Its cells carry too much EZHIP (a protein that blocks a normal "off switch" on gene packaging) and too little H3K27me3 (that off switch itself — a chemical tag on histones, the spools DNA wraps around).

The phase 2 trial (phase 2 = mid-stage testing of whether a treatment shows an effect) uses a clever window design: participants take metformin for about six weeks before their planned tumor surgery. Doctors then compare the removed tumor with the pre-treatment biopsy. The primary endpoint — the main question the trial must answer — is whether enough patients show a 10% drop in EZHIP and/or a 10% rise in H3K27me3\. A second phase measures the disease stabilization rate by RAPNO criteria (standard rules for judging brain-tumor scans in children).

The study is non-randomized and open-label (everyone gets metformin; no placebo group), aims for 30 participants, and runs at six US centers including UCSF and Johns Hopkins; completion is expected around 2030\. Context: pooled metformin trials in adults have been sobering — see [our review of 19 pooled metformin trials](https://www.sanarelab.science/metformin-chemo-adjuvant-meta-analysis-19-rcts-survival-2026/) — and for other repurposing attempts in children's brain tumors, see [another repurposing approach in pediatric brain tumors](https://www.sanarelab.science/mebendazole-ketogenic-diet-pediatric-glioma-2026/). Dosing questions: our [dosing calculator](https://www.sanarelab.science/protocol-dosing-workspace/).

## In plain words

**What happened:** Doctors at six US hospitals are testing the diabetes pill metformin in children and young adults whose PFA ependymoma — a brain tumor — came back after surgery and radiation.

**Why it matters:** This tumor grows because its gene switches are set wrong, not because of DNA damage. Metformin might flip some switches back; patients take it before surgery so doctors can check the removed tumor for changes.

**How strong is the evidence:** A registered phase 2 trial, now recruiting — there are no results yet. It is small (30 planned) with no placebo group — it can show hints, not proof.

**What it means for you:** If your child has recurrent PFA ependymoma, ask your oncology team about trial NCT07226453\. Do not start metformin on your own — it has real risks.

## References

1. Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma (phase 2, recruiting; trial record — no results yet). ClinicalTrials.gov. Record updated 2026 Oct 14\. [ClinicalTrials.gov: NCT07226453](https://clinicaltrials.gov/study/NCT07226453?ref=sanarelab.science).

## Medical Disclaimer

This article is informational, not medical advice. A trial registration describes a plan, not proven results. Never change medication without consulting your doctor.