Metformin for Brain Tumor
(PNOC041 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the effectiveness of metformin in treating a type of brain tumor called Posterior Fossa Group A (PFA) ependymoma in children and young adults. Researchers aim to determine if metformin can slow or stop the growth of tumors that have returned or worsened after previous treatments. Participants who have undergone surgery and radiation for PFA ependymoma but still experience recurring or progressing tumors may be suitable for this study. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group, providing participants an opportunity to contribute to significant findings.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications, but you cannot be on metformin or any investigational agents. If you're on dexamethasone or anti-seizure medication, you must be on a stable dose for at least a week before joining.
Is there any evidence suggesting that metformin is likely to be safe for humans?
Research has shown that metformin is usually well-tolerated. In a small study with 24 children and teens who underwent brain radiation treatment, metformin proved to be safe and manageable. Another study confirmed its safety for children who survived brain tumors. Additionally, the FDA has approved metformin for treating diabetes, further supporting its safety. However, like any treatment, it may cause side effects, so discussing these with the trial team is important.12345
Why do researchers think this study treatment might be promising for brain tumors?
Metformin is unique because it offers a potential new approach to treating brain tumors, specifically PFA ependymoma, by using a drug traditionally prescribed for diabetes. Unlike standard treatments like surgery and radiation, metformin is taken orally, which could make it a more convenient option for patients. Researchers are excited about metformin because it might work by influencing cancer cell metabolism, potentially slowing down tumor growth in a way that's different from existing therapies. This could offer new hope for patients whose tumors have not responded to traditional methods.
What evidence suggests that metformin might be an effective treatment for brain tumors?
Research has shown that metformin might help treat brain tumors by slowing their growth. In studies with aggressive brain tumors, metformin was linked to longer survival and slower tumor progression. It also appears to make tumor cells more responsive to treatments like chemotherapy and radiation. Metformin might target specific cancer cells that can cause tumors to recur, potentially preventing this. These early findings suggest that metformin could be effective for brain tumors like PFA ependymoma. Participants in this trial will receive metformin in either the Target Validation Phase or the Efficacy Phase, depending on their disease status and treatment history.12346
Who Is on the Research Team?
Sabine Mueller, MD, PhD
Principal Investigator
University of California, San Francisco
Santhosh A Upadhyaya, MD
Principal Investigator
University of Michigan, C. S. Mott Children's Hospital
Are You a Good Fit for This Trial?
This trial is for children and young adults aged 1-39 with recurrent or progressive PFA ependymoma, a type of brain tumor. They must have had prior surgery and radiation, controlled seizures if present, no diabetes or pre-diabetes, not pregnant or breastfeeding, and meet specific organ function criteria. Participants cannot be on metformin already or have uncontrolled illnesses.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Target Validation (TV) Phase
Participants receive daily oral metformin with a weekly increasing dosage schedule prior to planned surgery
Efficacy Phase
Participants receive daily oral metformin until disease progression or unacceptable adverse events
Follow-up
Participants are monitored for safety and effectiveness after treatment under the PNOC COMP protocol
What Are the Treatments Tested in This Trial?
Interventions
- Metformin
Trial Overview
The study tests the effects of Metformin on PFA ependymoma in patients who are undergoing surgical resection. It includes MRI and MRS imaging techniques along with specimen collection to assess the drug's impact on the tumor.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
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).
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).
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, San Francisco
Lead Sponsor
The Lilabean Foundation, Inc.
Collaborator
Pediatric Neuro-Oncology Consortium
Collaborator
Citations
Metformin as Potential Therapy for High-Grade Glioma - PMC
The use of metformin has been associated with better overall and progression-free survival of patients with high-grade glioma [9]. Repurposing metformin as ...
Metformin and glioma: Targeting metabolic dysregulation ...
Preclinical studies demonstrate metformin's efficacy in reducing tumor growth and enhancing the sensitivity of glioma cells to chemotherapy and radiotherapy.
Study Details | NCT05230758 | Effect of Metformin on ...
We hypothesize that 16 weeks of treatment with metformin will be associated with better cognitive outcomes than 16 weeks of treatment with placebo.
Advances in Anti-Cancer Drug Development: Metformin as ...
By targeting glioma stem cells, metformin may reduce tumor recurrence and enhance the overall effectiveness of TMZ and radiation therapy. Metformin can increase ...
Phase I/II study of maintenance therapy with metformin and ...
Our phase I findings indicated that MF at doses of up to 2,250 mg/day combined with maintenance TMZ was well tolerated (Cancers, 2022). Here, we ...
Assessment of cognitive and neural recovery in survivors of ...
Second, for our primary endpoint in the pilot trial, we found that metformin is safe to use and tolerable in survivors of pediatric brain tumors. While an ...
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