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

NCT05546476 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: C36510032023-510446-24-00
• Study Type: interventional
• Target: 187
• Locations: 11 countries
• Sites: 78

Brief Summary

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

Conditions

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

Keywords

cancer; anorexia; cachexia; weight loss; loss of appetite; fatigue

Outcome Measures

Primary Outcomes (1)

• Part A: Change From Baseline in Body Weight at Week 12

  Body weight was measured in kilograms using a calibrated weighing scale. Baseline was defined as the last average of the duplicate measurements prior to, or on Day 1. The average of the duplicate body weights collected at assessment time was considered. The posterior medians and 90 percent (%) credible intervals (5th and 95th percentiles of the relevant posterior distribution) were reported for each randomized dose (including placebo). 4-Parameter maximal effect (E max) model: change from baseline = E 0 + (E max \* dose\^Hill) / (ED 50\^Hill + dose\^Hill), where E0 is the placebo effect, E max is the maximum effect, ED 50 is the dose producing 50% of the maximum effect, and Hill is the slope parameter. Model utilized a Bayesian methodology with a robustified, informative meta-analytic predictive prior for the placebo change from baseline at week 12.

  Baseline, Week 12

Secondary Outcomes (12)

• Part A: Change From Baseline in Physical Activity at Week 12

  Baseline, Week 12

• Part A: Change From Baseline in Mean Activity Level During Maximum 6 Minutes at Week 12

  Baseline, Week 12

• Part A: Change From Baseline in Total Vector Magnitude at Week 12

  Baseline, Week 12

• Part A: Change From Baseline in Gait at Week 12

  Baseline, Week 12

• Part A: Change From Baseline in Functional Assessment of Anorexia-Cachexia Therapy- Anorexia and Cachexia Subscale (FAACT-ACS) at Week 12

  Baseline (prior to dose on Day 1), Week 12

Study Arms (4)

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

EXPERIMENTAL

ponsegromab low dose subcutaneous injection every 4 weeks

Drug: ponsegromab

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

PLACEBO COMPARATOR

Match placebo subcutaneous injection every 4 weeks

Drug: Placebo for ponsegromab

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

EXPERIMENTAL

ponsegromab medium dose subcutaneous injection every 4 weeks

Drug: ponsegromab

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

EXPERIMENTAL

ponsegromab high dose subcutaneous injection every 4 weeks

Drug: ponsegromab

Interventions

ponsegromab DRUG

Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment

Double-Blind ponsegromab Treatment high dose followed by Open Label ponsegromab Treatment; Double-Blind ponsegromab Treatment low dose followed by Open Label ponsegromab Treatment; Double-Blind ponsegromab Treatment medium dose followed by Open Label ponsegromab Treatment

Placebo for ponsegromab DRUG

Double-Blind placebo Treatment followed by Open Label ponsegromab Treatment

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (78)

CARTI Conway

Conway, Arkansas, 72034, United States

Location

CARTI Cancer Center

Little Rock, Arkansas, 72205, United States

Location

CARTI North Little Rock

North Little Rock, Arkansas, 72117, United States

Location

CARTI Stuttgart

Stuttgart, Arkansas, 72160, United States

Location

Pacific Cancer Medical Center INC

Anaheim, California, 92801, United States

Location

Beverly Hills Cancer Center

Beverly Hills, California, 90211, United States

Location

Emad Ibrahim,MD,INC.

Redlands, California, 92373, United States

Location

Providence Medical Foundation

Santa Rosa, California, 95403, United States

Location

IU Health Arnett Cancer Center

Lafayette, Indiana, 47904, United States

Location

Pennington Biomedical Research Center

Baton Rouge, Louisiana, 70808, United States

Location

Tandem Clinical Research

Marrero, Louisiana, 70072, United States

Location

Bozeman Health Deaconess Hospital

Bozeman, Montana, 59715, United States

Location

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

Portland, Oregon, 97239, United States

Location

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

Portland, Oregon, 97239, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Carta - Clinical Associates in Research Therapeutics of America, LLC

San Antonio, Texas, 78212, United States

Location

Virginia Mason Medical Center

Seattle, Washington, 98101, United States

Location

Wenatchee Valley Hospital

Wenatchee, Washington, 98801, United States

Location

St Vincent's Hospital Sydney

Darlinghurst, New South Wales, 2010, Australia

Location

Orange Hospital

Orange, New South Wales, 2800, Australia

Location

Peter MacCallum Cancer Centre

Melbourne, Victoria, 3000, Australia

Location

Western Health-Sunshine & Footscray Hospitals

St Albans, Victoria, 3021, Australia

Location

Complex Oncology Center - Burgas

Burgas, 8000, Bulgaria

Location

Specialized Hospital for Active Treatment of Oncology - Haskovo

Haskovo, 6300, Bulgaria

Location

Complex Oncology Center - Ruse EOOD

Rousse, 7002, Bulgaria

Location

Complex Oncology Center - Shumen

Shumen, 9700, Bulgaria

Location

Multiprofile Hospital for Active Treatment Serdika EOOD

Sofia, 1303, Bulgaria

Location

MHAT for Women's Health Nadezhda

Sofia, 1330, Bulgaria

Location

University Multiprofile Hospital for Active Treatment Sofiamed

Sofia, 1797, Bulgaria

Location

Complex Oncology Center - Vratsa

Vratsa, 3000, Bulgaria

Location

The Ottawa Hospital - General Campus

Ottawa, Ontario, K1H 8L6, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

CIUSSS- saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, G7H 5H6, Canada

Location

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

Rimouski, Quebec, G5L 5T1, Canada

Location

Peking University First Hospital

Beijing, Beijing Municipality, 100034, China

Location

Henan Cancer Hospital

Zhengzhou, Henan, 450008, China

Location

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

Wuhan, Hubei, 430030, China

Location

Changzhou No.2 People's Hospital

Changzhou, Jiangsu, 213003, China

Location

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, 330006, China

Location

Qilu Hospital of Shandong University

Jinan, Shandong, 250012, China

Location

Shanghai Changhai Hospital

Shanghai, Shanghai Municipality, 200433, China

Location

Shanxi Provincial Cancer Hospital

Taiyuan, Shanxi, 030013, China

Location

Sir Run Run Shaw Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310016, China

Location

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

Wenzhou, Zhejiang, 325035, China

Location

Bacs-Kiskun Varmegyei Oktatokorhaz

Kecskemét, Bács-Kiskun county, 6000, Hungary

Location

Jász-Nagykun-Szolnok Vármegyei Hetényi Géza Kórház

Szolnok, Jász-Nagykun-Szolnok, 5004, Hungary

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

Országos Korányi Pulmonológiai Intézet

Budapest, 1121, Hungary

Location

National Cancer Center Hospital East

Kashiwa, Chiba, 277-8577, Japan

Location

National Hospital Organization Shikoku Cancer Center

Matsuyama, Ehime, 791-0280, Japan

Location

Hyogo Cancer Center

Akashi, Hyōgo, 673-8558, Japan

Location

Kanagawa cancer center

Yokohama, Kanagawa, 241-8515, Japan

Location

Aichi Cancer Center Hospital

Nagoya, Nagoya, Aichi, 464-8681, Japan

Location

Shizuoka Cancer Center

Nagaizumi-cho, Shizuoka, 411-8777, Japan

Location

University Hospital,Kyoto Prefectural University of Medicine

Kyoto, 602-8566, Japan

Location

The Cancer Institute Hospital of JFCR

Tokyo, 135-8550, Japan

Location

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

Krakow, Lesser Poland Voivodeship, 30-348, Poland

Location

Regionalny Szpital Specjalistyczny im. Dr. Wladyslawa Bieganskiego

Grudziądz, 86-300, Poland

Location

NZOZ "Vegamed"

Katowice, 40-060, Poland

Location

Specjalistyczna Praktyka Lekarska Slawomir Mandziuk

Lublin, 20-093, Poland

Location

Fakultna nemocnica s poliklinikou F. D. Roosevelta Banska Bystrica

Banská Bystrica, 975 17, Slovakia

Location

Univerzitna nemocnica Bratislava, Nemocnica Ruzinov

Bratislava, 826 06, Slovakia

Location

Narodny onkologicky ustav, II. Onkologicka klinika LFUK a NOU

Bratislava, 833 10, Slovakia

Location

Fakultna nemocnica s poliklinikou Nove Zamky

Nové Zámky, 940 34, Slovakia

Location

Nemocnica na okraji mesta, n.o.

Partizánske, 95801, Slovakia

Location

Fakultna nemocnica Trnava

Trnava, 917 75, Slovakia

Location

Hospital Universitari General de Catalunya

Sant Cugat del Vallès, Barcelona [barcelona], 08915, Spain

Location

Institut Català d'Oncologia - L'Hospitalet

L'Hospitalet de Llobregat, Catalunya [cataluña], 08908, Spain

Location

Hospital Son Llàtzer

Palma, Illes Balears [islas Baleares], 07198, Spain

Location

Fundación Instituto Valenciano de Oncología

Valencia, Valenciana, Comunitat, 46009, Spain

Location

Hospital Universitario HM Sanchinarro

Madrid, 28050, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain

Location

Chi Mei Hospital - Liouying Branch

Tainan, Tainan, 73657, Taiwan

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, 80756, Taiwan

Location

China Medical University Hospital

Taichung, 404332, Taiwan

Location

National Cheng Kung University Hospital

Tainan, 704, Taiwan

Location

Chang Gung Medical Foundation-Linkou Branch

Taoyuan District, 333, Taiwan

Location

Related Publications (2)

• 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.

  PMID: 39282907 DERIVED

• 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.

  PMID: 38500292 DERIVED

Related Links

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

MeSH Terms

Conditions

Carcinoma, Non-Small-Cell Lung; Pancreatic Neoplasms; Colorectal Neoplasms; Anorexia; Fatigue; Cachexia; Neoplasms; Weight Loss

Condition Hierarchy (Ancestors)

Carcinoma, Bronchogenic; Bronchial Neoplasms; Lung Neoplasms; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Lung Diseases; Respiratory Tract Diseases; Digestive System Neoplasms; Endocrine Gland Neoplasms; Digestive System Diseases; Pancreatic Diseases; Endocrine System Diseases; Intestinal Neoplasms; Gastrointestinal Neoplasms; Gastrointestinal Diseases; Colonic Diseases; Intestinal Diseases; Rectal Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Body Weight Changes; Body Weight; Thinness

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind for 12-week double-blind treatment period followed by an up to 1 year optional open-label treatment period
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2022
• First Posted: September 19, 2022
• Study Start: November 21, 2022
• Primary Completion: March 13, 2024
• Study Completion: April 23, 2025
• Last Updated: May 31, 2025
• Results First Posted: April 29, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share

Locations

ES (6); AU (4); PL (4); US (19); SL (6); BU (8); JP (8); HU (4); CA (4); TW (5); CN (10)