NCT05546476

Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15

A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO INVESTIGATE THE EFFICACY, SAFETY AND TOLERABILITY OF PONSEGROMAB IN PATIENTS WITH CANCER, CACHEXIA, AND ELEVATED CONCENTRATIONS OF GDF-15, FOLLOWED BY AN OPTIONAL OPEN-LABEL TREATMENT PERIOD (PROACC -1)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 187 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study to evaluate the efficacy, safety and tolerability of ponsegromab compared to placebo in patients with cancer, cachexia, and elevated GDF 15.

Detailed description

A 12 week double blind study to evaluate the efficacy, safety and tolerability of ponsegromab compared to placebo in patients with cancer, cachexia, and elevated GDF 15. During the initial 12-week treatment period (Part A), a total of 3 doses of ponsegromab or placebo will be administered 4 weeks apart subcutaneously. Each dose contains two injections. Part B is an optional open-label treatment period consisting of ponsegromab administered every 4 weeks subcutaneously for up to one year. Part B does not include placebo. Assessments include: * Body weight measurements * Measure the impact of ponsegromab compared to placebo on physical activity. * Measure the impact of ponsegromab compared to placebo on appetite, fatigue, nausea, vomiting and physical function questionnaires. * Blood samples to evaluate safety and additional endpoints including the amount of study drug in the blood and the effects of the study drug on levels of GDF15 * Up to 3 additional blood samples (two samples during Part A and one sample during Part B, if relevant) in a subset of participants as part of a substudy for more comprehensive assessment of the amount of study drug in the blood and of the effects of the study drug on levels of GDF-15.

Conditions

• Non-small Cell Lung Cancer
• Pancreatic Cancer
• Colorectal Cancer
• Loss of Appetite
• Fatigue
• Cachexia

Interventions

Type	Name	Description
DRUG	ponsegromab	Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment
DRUG	Placebo for ponsegromab	Double-Blind placebo Treatment followed by Open Label ponsegromab Treatment

Timeline

• Start date: 2022-11-21
• Primary completion: 2024-03-13
• Completion: 2025-04-23
• First posted: 2022-09-19
• Last updated: 2025-05-31
• Results posted: 2025-04-29

Locations

78 sites across 11 countries: United States, Australia, Bulgaria, Canada, China, Hungary, Japan, Poland, Slovakia, Spain, Taiwan

Regulatory

• FDA-regulated drug study