NCT05183204

Paxalisib With a High Fat, Low Carb Diet and Metformin for Glioblastoma

A Phase 2 Trial of Paxalisib Combined With a Ketogenic Diet and Metformin for Newly Diagnosed and Recurrent Glioblastoma

• Status: Suspended
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (estimated)
• Sponsor: Weill Medical College of Cornell University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is for patients with newly diagnosed glioblastoma, as well as patients who have recurring glioblastoma. Subjects will be given daily paxalisib and metformin while also maintaining a ketogenic diet. The purpose of this study is to assess the safety of Paxalisib while maintaining a ketogenic diet (a high fat, low carbohydrate diet) and Metformin (a drug approved by the Food and Drug Administration to treat type 2 diabetes), and to see what effects it has on glioblastoma.

Detailed description

This is a two stage, two cohort phase 2 trial of a new blood-brain penetrant PI3K/mTOR inhibitor (paxalisib) combined with a ketogenic diet plus metformin in patients with either newly diagnosed MGMT unmethylated glioblastoma or patients with recurrent glioblastoma regardless of MGMT promoter methylation status.

Conditions

• Glioblastoma

Interventions

Type	Name	Description
DRUG	Paxalisib	Patients will receive paxalisib starting at a dose of 45 mg/day. If well tolerated after 28 days, the dose of paxalisib will be increased to 60 mg/day.
DRUG	Metformin	Patients will receive metformin on Cycle 1, Day 1 at a starting dose of 850 mg QD, and if tolerated, will be increased to 850 mg BID on Cycle 2, Day 1 (1700 mg/day). If that dose is tolerated, metformin will be increased to 850 mg TID (2550 mg/day) beginning on Cycle 3, Day 1.
OTHER	Ketogenic Diet	The ketogenic diet is high-fat, low carbohydrate diet. Ketogenic diet will be maintained on a continuous basis starting on Cycle 1, Day 1 and continuing throughout the trial.

Timeline

• Start date: 2022-02-14
• Primary completion: 2026-06-01
• Completion: 2026-06-01
• First posted: 2022-01-10
• Last updated: 2025-12-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study