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

NCT ID: NCT05546476

Last Updated: 2025-05-31

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

187 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-21

Study Completion Date

2025-04-23

Brief Summary

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

Detailed Description

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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

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double-blind for 12-week double-blind treatment period followed by an up to 1 year optional open-label treatment period

Study Groups

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

ponsegromab low dose subcutaneous injection every 4 weeks

Group Type EXPERIMENTAL

ponsegromab

Intervention Type DRUG

Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment

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

Match placebo subcutaneous injection every 4 weeks

Group Type PLACEBO_COMPARATOR

Placebo for ponsegromab

Intervention Type DRUG

Double-Blind placebo Treatment followed by Open Label ponsegromab Treatment

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

ponsegromab medium dose subcutaneous injection every 4 weeks

Group Type EXPERIMENTAL

ponsegromab

Intervention Type DRUG

Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment

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

ponsegromab high dose subcutaneous injection every 4 weeks

Group Type EXPERIMENTAL

ponsegromab

Intervention Type DRUG

Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment

Interventions

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ponsegromab

Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment

Intervention Type DRUG

Placebo for ponsegromab

Double-Blind placebo Treatment followed by Open Label ponsegromab Treatment

Intervention Type DRUG

Eligibility Criteria

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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.

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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CARTI Conway

Conway, Arkansas, United States

Site Status

CARTI Cancer Center

Little Rock, Arkansas, United States

Site Status

CARTI North Little Rock

North Little Rock, Arkansas, United States

Site Status

CARTI Stuttgart

Stuttgart, Arkansas, United States

Site Status

Pacific Cancer Medical Center INC

Anaheim, California, United States

Site Status

Beverly Hills Cancer Center

Beverly Hills, California, United States

Site Status

Emad Ibrahim,MD,INC.

Redlands, California, United States

Site Status

Providence Medical Foundation

Santa Rosa, California, United States

Site Status

IU Health Arnett Cancer Center

Lafayette, Indiana, United States

Site Status

Pennington Biomedical Research Center

Baton Rouge, Louisiana, United States

Site Status

Tandem Clinical Research

Marrero, Louisiana, United States

Site Status

Bozeman Health Deaconess Hospital

Bozeman, Montana, United States

Site Status

Oregon Health and Science University: Center for Health and Healing 1

Portland, Oregon, United States

Site Status

Oregon Health and Science University: Center for Health and Healing 2

Portland, Oregon, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Carta - Clinical Associates in Research Therapeutics of America, LLC

San Antonio, Texas, United States

Site Status

Virginia Mason Medical Center

Seattle, Washington, United States

Site Status

Wenatchee Valley Hospital

Wenatchee, Washington, United States

Site Status

St Vincent's Hospital Sydney

Darlinghurst, New South Wales, Australia

Site Status

Orange Hospital

Orange, New South Wales, Australia

Site Status

Peter MacCallum Cancer Centre

Melbourne, Victoria, Australia

Site Status

Western Health-Sunshine & Footscray Hospitals

St Albans, Victoria, Australia

Site Status

Complex Oncology Center - Burgas

Burgas, , Bulgaria

Site Status

Specialized Hospital for Active Treatment of Oncology - Haskovo

Haskovo, , Bulgaria

Site Status

Complex Oncology Center - Ruse EOOD

Rousse, , Bulgaria

Site Status

Complex Oncology Center - Shumen

Shumen, , Bulgaria

Site Status

Multiprofile Hospital for Active Treatment Serdika EOOD

Sofia, , Bulgaria

Site Status

MHAT for Women's Health Nadezhda

Sofia, , Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment Sofiamed

Sofia, , Bulgaria

Site Status

Complex Oncology Center - Vratsa

Vratsa, , Bulgaria

Site Status

The Ottawa Hospital - General Campus

Ottawa, Ontario, Canada

Site Status

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Site Status

CIUSSS- saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada

Site Status

Centre intégré de santé et de services sociaux du Bas Saint-Laurent- Hôpital régional de Rimouski

Rimouski, Quebec, Canada

Site Status

Peking University First Hospital

Beijing, Beijing Municipality, China

Site Status

Henan Cancer Hospital

Zhengzhou, Henan, China

Site Status

Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, China

Site Status

Changzhou No.2 People's Hospital

Changzhou, Jiangsu, China

Site Status

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, China

Site Status

Qilu Hospital of Shandong University

Jinan, Shandong, China

Site Status

Shanghai Changhai Hospital

Shanghai, Shanghai Municipality, China

Site Status

Shanxi Provincial Cancer Hospital

Taiyuan, Shanxi, China

Site Status

Sir Run Run Shaw Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Site Status

The 2nd Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University

Wenzhou, Zhejiang, China

Site Status

Bacs-Kiskun Varmegyei Oktatokorhaz

Kecskemét, Bács-Kiskun county, Hungary

Site Status

Jász-Nagykun-Szolnok Vármegyei Hetényi Géza Kórház

Szolnok, Jász-Nagykun-Szolnok, Hungary

Site Status

Semmelweis Egyetem

Budapest, , Hungary

Site Status

Országos Korányi Pulmonológiai Intézet

Budapest, , Hungary

Site Status

National Cancer Center Hospital East

Kashiwa, Chiba, Japan

Site Status

National Hospital Organization Shikoku Cancer Center

Matsuyama, Ehime, Japan

Site Status

Hyogo Cancer Center

Akashi, Hyōgo, Japan

Site Status

Kanagawa cancer center

Yokohama, Kanagawa, Japan

Site Status

Aichi Cancer Center Hospital

Nagoya, Nagoya, Aichi, Japan

Site Status

Shizuoka Cancer Center

Nagaizumi-cho, Shizuoka, Japan

Site Status

University Hospital,Kyoto Prefectural University of Medicine

Kyoto, , Japan

Site Status

The Cancer Institute Hospital of JFCR

Tokyo, , Japan

Site Status

Centrum Badań Klinicznych Jagiellońskie Centrum Innowacji sp. z o.o.

Krakow, Lesser Poland Voivodeship, Poland

Site Status

Regionalny Szpital Specjalistyczny im. Dr. Wladyslawa Bieganskiego

Grudziądz, , Poland

Site Status

NZOZ "Vegamed"

Katowice, , Poland

Site Status

Specjalistyczna Praktyka Lekarska Slawomir Mandziuk

Lublin, , Poland

Site Status

Fakultna nemocnica s poliklinikou F. D. Roosevelta Banska Bystrica

Banská Bystrica, , Slovakia

Site Status

Univerzitna nemocnica Bratislava, Nemocnica Ruzinov

Bratislava, , Slovakia

Site Status

Narodny onkologicky ustav, II. Onkologicka klinika LFUK a NOU

Bratislava, , Slovakia

Site Status

Fakultna nemocnica s poliklinikou Nove Zamky

Nové Zámky, , Slovakia

Site Status

Nemocnica na okraji mesta, n.o.

Partizánske, , Slovakia

Site Status

Fakultna nemocnica Trnava

Trnava, , Slovakia

Site Status

Hospital Universitari General de Catalunya

Sant Cugat del Vallès, Barcelona [barcelona], Spain

Site Status

Institut Català d'Oncologia - L'Hospitalet

L'Hospitalet de Llobregat, Catalunya [cataluña], Spain

Site Status

Hospital Son Llàtzer

Palma, Illes Balears [islas Baleares], Spain

Site Status

Fundación Instituto Valenciano de Oncología

Valencia, Valenciana, Comunitat, Spain

Site Status

Hospital Universitario HM Sanchinarro

Madrid, , Spain

Site Status

Hospital Universitari i Politecnic La Fe

Valencia, , Spain

Site Status

Chi Mei Hospital - Liouying Branch

Tainan City, Tainan, Taiwan

Site Status

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, , Taiwan

Site Status

China Medical University Hospital

Taichung, , Taiwan

Site Status

National Cheng Kung University Hospital

Tainan City, , Taiwan

Site Status

Chang Gung Medical Foundation-Linkou Branch

Taoyuan District, , Taiwan

Site Status

Countries

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United States Australia Bulgaria Canada China Hungary Japan Poland Slovakia Spain Taiwan

References

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Groarke JD, Crawford J, Collins SM, Lubaczewski S, Roeland EJ, Naito T, Hendifar AE, Fallon M, Takayama K, Asmis T, Dunne RF, Karahanoglu I, Northcott CA, Harrington MA, Rossulek M, Qiu R, Saxena AR. Ponsegromab for the Treatment of Cancer Cachexia. N Engl J Med. 2024 Dec 19;391(24):2291-2303. doi: 10.1056/NEJMoa2409515. Epub 2024 Sep 14.

Reference Type DERIVED
PMID: 39282907 (View on PubMed)

Groarke JD, Crawford J, Collins SM, Lubaczewski SL, Breen DM, Harrington MA, Jacobs I, Qiu R, Revkin J, Rossulek MI, Saxena AR. Phase 2 study of the efficacy and safety of ponsegromab in patients with cancer cachexia: PROACC-1 study design. J Cachexia Sarcopenia Muscle. 2024 Jun;15(3):1054-1061. doi: 10.1002/jcsm.13435. Epub 2024 Mar 18.

Reference Type DERIVED
PMID: 38500292 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=C3651003

To obtain contact information for a study center near you, click here.

Other Identifiers

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2023-510446-24-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

C3651003

Identifier Type: -

Identifier Source: org_study_id

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