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

Sponsor

Pfizer (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05546476

Collaborator

(none)

168

Enrollment

Locations

Arms

18.2

Anticipated Duration (Months)

Patients Per Site

4.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Non-small Cell Lung Cancer Pancreatic Cancer Colorectal Cancer Loss of Appetite Fatigue Cachexia	Drug: ponsegromab Drug: Placebo for ponsegromab	Phase 2

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, nausea and fatigue 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 GDF-15

Study Design

Study Type:

Interventional

Anticipated Enrollment :

168 participants

Allocation:

Randomized

Intervention Model:

Sequential Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

Double-blind for 12-week double-blind treatment period followed by an up to 1 year optional open-label treatment period

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Sep 9, 2022

Anticipated Primary Completion Date :

Mar 15, 2024

Anticipated Study Completion Date :

Mar 15, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Double-Blind ponsegromab Treatment low dose followed by Open Label ponsegromab Treatment ponsegromab 100 mg subcutaneous injection every 4 weeks	Drug: ponsegromab Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment
Placebo Comparator: Double-Blind Placebo Treatment followed by Open-Label ponsegromab Treatment Match placebo subcutaneous injection every 4 weeks	Drug: Placebo for ponsegromab Double-Blind placebo Treatment followed by Open Label ponsegromab Treatment
Experimental: Double-Blind ponsegromab Treatment medium dose followed by Open Label ponsegromab Treatment ponsegromab 200 mg subcutaneous injection every 4 weeks	Drug: ponsegromab Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment
Experimental: Double-Blind ponsegromab Treatment high dose followed by Open Label ponsegromab Treatment ponsegromab 400 mg subcutaneous injection every 4 weeks	Drug: ponsegromab Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment

Outcome Measures

Primary Outcome Measures

Change from baseline in body weight for ponsegromab compared to placebo [baseline, Week 12]

Secondary Outcome Measures

Change from baseline in physical activity as measured with remote digital sensors [baseline, Week 12]
evaluation of physical activity (Sedentary, light and moderate) and gait speed.
change from baseline in Functional Assessment of Anorexia-Cachexia Therapy (FAACT-ACS and FAACT-5IASS) scores [baseline, Week 12]
FACT-G score sums 27 items; ACS score sums 12 items; both use a 5-point scale (0-4).Higher scores are associated with a higher health-related quality of life.
Incidence of adverse events [Week 12]
Incidence of lab abnormalities [Week 12]
Incidence of vital sign abnormalities [Week 12]
Incidence of ECG abnormalities [Week 12]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Documented diagnosis of non-small cell lung, pancreatic, colorectal cancer
Cachexia defined by Fearon criteria
Serum GDF-15 concentrations
Signed informed consent

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 renal or liver function

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Carta - Clinical Associates in Research Therapeutics of America, LLC	San Antonio	Texas	United States	78212
2	Wenatchee Valley Hospital	Wenatchee	Washington	United States	98801