NCT04750941

Study of Copanlisib and Ketogenic Diet

Pilot Phase II Study of the PI3K Inhibitor Copanlisib in Combination With a Ketogenic Diet in the Treatment of Patients With Relapsed or Refractory Follicular Lymphoma or Endometrial Cancer

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Columbia University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, open label, pilot phase II study of the PI3K inhibitor copanlisib in combination with a ketogenic diet in the treatment of patients with one of the following malignancies: (a) relapsed or refractory (R/R) follicular lymphoma (FL), (b) R/R endometrial cancer (EC) with a documented activating mutation in PIK3CA or loss of phosphatase and tensin homolog (PTEN).

Detailed description

As the investigators recently reported ketogenic diet can suppress hyperinsulinemia associated with PI3K inhibitors, leading to potentiation of the anti-tumor effects of PI3K inhibitors. Copanlisib potently inhibits PI3Kα and PI3Kδ. It has been approved for the treatment of relapsed follicular lymphoma, based on ORR of 59% (84 of 142 patients). The CR rate in FL was 14%, and the median progression-free survival was 11.2 months. Copanlisib demonstrated encouraging clinical activity in marginal zone lymphoma (ORR 70% including 9% CR). While these results are clinically meaningful, FL and MZL inevitably develop resistance to copanlisib with time, even in those patients who initially respond to the therapy. Novel strategies to improve the efficacy of copanlisib in FL and MZL, by improving CR and PFS, may transform how to manage these incurable malignancies.

Conditions

• Follicular Lymphoma
• Endometrial Cancer

Interventions

Type	Name	Description
DRUG	Copanlisib	Copanlisib will be infused intravenously on days 1, 8, 15 of each cycle, over 1 hour, of 28-day cycles.
OTHER	Ketogenic Diet	In cycle 1, patients will first start the ketogenic diet for 7 days (Day -6 to Day 0). Only the patients who demonstrate compliance and tolerance with the ketogenic diet, as confirmed by pertinent blood and urine tests and a diary of diet, will be allowed to continue the study and start copanlisib on Day 1. The ketogenic diet will continue daily throughout the treatment days. In cycle 2 and beyond, patients will start the ketogenic diet and copanlisib on day 1. The ketogenic diet will then continue daily throughout the treatment days.

Timeline

• Start date: 2022-02-10
• Primary completion: 2023-06-09
• Completion: 2023-06-09
• First posted: 2021-02-11
• Last updated: 2025-03-04
• Results posted: 2025-03-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study