Clinical trial NCT05546476: Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15
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 (PROACC -1)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-09-19
Enrollment: 187
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.

Eligibility 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

Primary outcomes:
measure: Part A: Change From Baseline in Body Weight at Week 12
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Part A: Change From Baseline in Physical Activity at Week 12
timeFrame: Baseline, Week 12
measure: Part A: Change From Baseline in Mean Activity Level During Maximum 6 Minutes at Week 12
timeFrame: Baseline, Week 12
measure: Part A: Change From Baseline in Total Vector Magnitude at Week 12
timeFrame: Baseline, Week 12
measure: Part A: Change From Baseline in Gait at Week 12
timeFrame: Baseline, Week 12
measure: Part A: Change From Baseline in Functional Assessment of Anorexia-Cachexia Therapy- Anorexia and Cachexia Subscale (FAACT-ACS) at Week 12
timeFrame: Baseline (prior to dose on Day 1), Week 12
measure: Part A: Change From Baseline in FAACT- 5-Item Anorexia Symptom Scale (5IASS) at Week 12
timeFrame: Baseline (prior to dose on Day 1), Week 12
measure: Part A: Change From Baseline in Cancer-Related Cachexia Symptom Diary (CRCSD) Scores at Week 12: Appetite, Nausea and Physical Fatigue
timeFrame: Baseline, Week 12
measure: Part A: Median Change From Baseline in CRCSD Scores at Week 12: Vomiting Frequency
timeFrame: Baseline, Week 12
measure: Part A: Number of Participants With Treatment Emergent Adverse Events (TEAE)
timeFrame: From start of study drug on Day 1 maximum up to 4 weeks post last dose on Week 12 (maximum up to approximately Week 16)
measure: Part A: Number of Participants With Incidence of Laboratory Test Abnormalities
timeFrame: Day 1 up to Week 12
measure: Part A: Number of Participants With Post-Baseline Vital Signs Meeting the Predefined Criteria
timeFrame: Day 1 up to Week 12
measure: Part A: Number of Participants With Clinically Significant Echocardiogram (ECG) Abnormalities
timeFrame: Day 1 up to Week 12
Other outcomes:
Locations:
city: Conway
country: United States
facility: CARTI Conway
state: Arkansas
city: Little Rock
country: United States
facility: CARTI Cancer Center
state: Arkansas
city: North Little Rock
country: United States
facility: CARTI North Little Rock
state: Arkansas
city: Stuttgart
country: United States
facility: CARTI Stuttgart
state: Arkansas
city: Anaheim
country: United States
facility: Pacific Cancer Medical Center INC
state: California
city: Beverly Hills
country: United States
facility: Beverly Hills Cancer Center
state: California
city: Redlands
country: United States
facility: Emad Ibrahim,MD,INC.
state: California
city: Santa Rosa
country: United States
facility: Providence Medical Foundation
state: California
city: Lafayette
country: United States
facility: IU Health Arnett Cancer Center
state: Indiana
city: Baton Rouge
country: United States
facility: Pennington Biomedical Research Center
state: Louisiana
city: Marrero
country: United States
facility: Tandem Clinical Research
state: Louisiana
city: Bozeman
country: United States
facility: Bozeman Health Deaconess Hospital
state: Montana
city: Portland
country: United States
facility: Oregon Health and Science University: Center for Health and Healing 1
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University: Center for Health and Healing 2
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Houston
country: United States
facility: The University of Texas MD Anderson Cancer Center
state: Texas
city: San Antonio
country: United States
facility: Carta - Clinical Associates in Research Therapeutics of America, LLC
state: Texas
city: Seattle
country: United States
facility: Virginia Mason Medical Center
state: Washington
city: Wenatchee
country: United States
facility: Wenatchee Valley Hospital
state: Washington
city: Darlinghurst
country: Australia
facility: St Vincent's Hospital Sydney
state: New South Wales
city: Orange
country: Australia
facility: Orange Hospital
state: New South Wales
city: Melbourne
country: Australia
facility: Peter MacCallum Cancer Centre
state: Victoria
city: St Albans
country: Australia
facility: Western Health-Sunshine & Footscray Hospitals
state: Victoria
city: Burgas
country: Bulgaria
facility: Complex Oncology Center - Burgas
city: Haskovo
country: Bulgaria
facility: Specialized Hospital for Active Treatment of Oncology - Haskovo
city: Ruse
country: Bulgaria
facility: Complex Oncology Center - Ruse EOOD
city: Shumen
country: Bulgaria
facility: Complex Oncology Center - Shumen
city: Sofia
country: Bulgaria
facility: Multiprofile Hospital for Active Treatment Serdika EOOD
city: Sofia
country: Bulgaria
facility: MHAT for Women's Health Nadezhda
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment Sofiamed
city: Vratsa
country: Bulgaria
facility: Complex Oncology Center - Vratsa
city: Ottawa
country: Canada
facility: The Ottawa Hospital - General Campus
state: Ontario
city: Toronto
country: Canada
facility: Princess Margaret Cancer Centre
state: Ontario
city: Chicoutimi
country: Canada
facility: CIUSSS- saguenay-Lac-Saint-Jean
state: Quebec
city: Rimouski
country: Canada
facility: Centre intégré de santé et de services sociaux du Bas Saint-Laurent- Hôpital régional de Rimouski
state: Quebec
city: Beijing
country: China
facility: Peking University First Hospital
state: Beijing
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology
state: Hubei
city: Changzhou
country: China
facility: Changzhou No.2 People's Hospital
state: Jiangsu
city: Nanchang
country: China
facility: The First Affiliated Hospital of Nanchang University
state: Jiangxi
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shandong
city: Shanghai
country: China
facility: Shanghai Changhai Hospital
state: Shanghai
city: Taiyuan
country: China
facility: Shanxi Provincial Cancer Hospital
state: Shanxi
city: Hangzhou
country: China
facility: Sir Run Run Shaw Hospital of Zhejiang University School of Medicine
state: Zhejiang
city: Wenzhou
country: China
facility: The 2nd Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University
state: Zhejiang
city: Kecskemét
country: Hungary
facility: Bacs-Kiskun Varmegyei Oktatokorhaz
state: Bács-kiskun
city: Szolnok
country: Hungary
facility: Jász-Nagykun-Szolnok Vármegyei Hetényi Géza Kórház
state: Jász-nagykun-szolnok
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Budapest
country: Hungary
facility: Országos Korányi Pulmonológiai Intézet
city: Kashiwa
country: Japan
facility: National Cancer Center Hospital East
state: Chiba
city: Matsuyama
country: Japan
facility: National Hospital Organization Shikoku Cancer Center
state: Ehime
city: Akashi
country: Japan
facility: Hyogo Cancer Center
state: Hyogo
city: Yokohama
country: Japan
facility: Kanagawa cancer center
state: Kanagawa
city: Nagoya
country: Japan
facility: Aichi Cancer Center Hospital
state: Nagoya, Aichi
city: Nagaizumi-cho
country: Japan
facility: Shizuoka Cancer Center
state: Shizuoka
city: Kyoto
country: Japan
facility: University Hospital,Kyoto Prefectural University of Medicine
city: Tokyo
country: Japan
facility: The Cancer Institute Hospital of JFCR
city: Kraków
country: Poland
facility: Centrum Badań Klinicznych Jagiellońskie Centrum Innowacji sp. z o.o.
state: Małopolskie
city: Grudziadz
country: Poland
facility: Regionalny Szpital Specjalistyczny im. Dr. Wladyslawa Bieganskiego
city: Katowice
country: Poland
facility: NZOZ "Vegamed"
city: Lublin
country: Poland
facility: Specjalistyczna Praktyka Lekarska Slawomir Mandziuk
city: Banska Bystrica
country: Slovakia
facility: Fakultna nemocnica s poliklinikou F. D. Roosevelta Banska Bystrica
city: Bratislava
country: Slovakia
facility: Univerzitna nemocnica Bratislava, Nemocnica Ruzinov
city: Bratislava
country: Slovakia
facility: Narodny onkologicky ustav, II. Onkologicka klinika LFUK a NOU
city: Nove Zamky
country: Slovakia
facility: Fakultna nemocnica s poliklinikou Nove Zamky
city: Partizanske
country: Slovakia
facility: Nemocnica na okraji mesta, n.o.
city: Trnava
country: Slovakia
facility: Fakultna nemocnica Trnava
city: Sant Cugat del Vallès
country: Spain
facility: Hospital Universitari General de Catalunya
state: Barcelona [barcelona]
city: L'Hospitalet de Llobregat
country: Spain
facility: Institut Català d'Oncologia - L'Hospitalet
state: Catalunya [cataluña]
city: Palma
country: Spain
facility: Hospital Son Llàtzer
state: Illes Balears [islas Baleares]
city: Valencia
country: Spain
facility: Fundación Instituto Valenciano de Oncología
state: Valenciana, Comunitat
city: Madrid
country: Spain
facility: Hospital Universitario HM Sanchinarro
city: València
country: Spain
facility: Hospital Universitari i Politecnic La Fe
city: Tainan City
country: Taiwan
facility: Chi Mei Hospital - Liouying Branch
state: Tainan
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Chung-Ho Memorial Hospital
city: Taichung
country: Taiwan
facility: China Medical University Hospital
city: Tainan
country: Taiwan
facility: National Cheng Kung University Hospital
city: Taoyuan
country: Taiwan
facility: Chang Gung Medical Foundation-Linkou Branch
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: