Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15 (PROACC-1)

Pfizer

Status and phase: Active, not recruiting; Phase 2

Conditions: Loss of Appetite; Non-small Cell Lung Cancer; Cachexia; Fatigue; Colorectal Cancer; Pancreatic Cancer

Treatments: Drug: Placebo for ponsegromab; Drug: ponsegromab

Study type: Interventional

Funder types: Industry

Identifiers

NCT05546476

2022-003016-87 (EudraCT Number)

C3651003

Details and patient eligibility

About

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

Full 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.

Enrollment: 187 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Documented active diagnosis of non-small cell lung, pancreatic, colorectal cancer
• Cachexia defined by Fearon criteria of weight loss
• Serum GDF-15 concentrations
• Signed informed consent
• ECOG PS ≤3 with life expectancy of at least 4 months to be able to complete Part A.

Key Exclusion Criteria:

• Receiving tube feedings or parenteral nutrition at the time of Screening or Randomization.
• Current active reversible causes of decreased food intake.
• Cachexia caused by other reasons.
• History of allergic or anaphylactic reaction to any therapeutic or diagnostic monoclonal antibody.
• inadequate liver function
• renal disease requiring dialysis

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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187 participants in 4 patient groups, including a placebo group

Double-Blind ponsegromab Treatment low dose followed by Open Label ponsegromab Treatment

Experimental group

Description:

ponsegromab low dose subcutaneous injection every 4 weeks

Treatment:

Drug: ponsegromab

Double-Blind Placebo Treatment followed by Open-Label ponsegromab Treatment

Placebo Comparator group

Description:

Match placebo subcutaneous injection every 4 weeks

Treatment:

Drug: Placebo for ponsegromab

Double-Blind ponsegromab Treatment medium dose followed by Open Label ponsegromab Treatment

Experimental group

Description:

ponsegromab medium dose subcutaneous injection every 4 weeks

Treatment:

Drug: ponsegromab

Double-Blind ponsegromab Treatment high dose followed by Open Label ponsegromab Treatment

Experimental group

Description:

ponsegromab high dose subcutaneous injection every 4 weeks

Treatment:

Drug: ponsegromab