Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
A database of clinical trials and their results from Australia, New Zealand, and other countries.
account_circle
Log in
to register or update your trial
search
Search for trials
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05546476
Registration number
NCT05546476
Ethics application status
Date submitted
12/08/2022
Date registered
19/09/2022
Date last updated
31/05/2025
Titles & IDs
Public title
Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15
Query!
Scientific 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)
Query!
Secondary ID [1]
0
0
2023-510446-24-00
Query!
Secondary ID [2]
0
0
C3651003
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
PROACC-1
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Non-small Cell Lung Cancer
0
0
Query!
Pancreatic Cancer
0
0
Query!
Colorectal Cancer
0
0
Query!
Loss of Appetite
0
0
Query!
Fatigue
0
0
Query!
Cachexia
0
0
Query!
Condition category
Condition code
Diet and Nutrition
0
0
0
0
Query!
Other diet and nutrition disorders
Query!
Mental Health
0
0
0
0
Query!
Eating disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - ponsegromab
Treatment: Drugs - Placebo for ponsegromab
Experimental: Double-Blind ponsegromab Treatment low dose followed by Open Label ponsegromab Treatment - ponsegromab low dose subcutaneous injection every 4 weeks
Placebo comparator: Double-Blind Placebo Treatment followed by Open-Label ponsegromab Treatment - Match placebo subcutaneous injection every 4 weeks
Experimental: Double-Blind ponsegromab Treatment medium dose followed by Open Label ponsegromab Treatment - ponsegromab medium dose subcutaneous injection every 4 weeks
Experimental: Double-Blind ponsegromab Treatment high dose followed by Open Label ponsegromab Treatment - ponsegromab high dose subcutaneous injection every 4 weeks
Treatment: Drugs: ponsegromab
Double-Blind ponsegromab Treatment followed by Open Label ponsegromab Treatment
Treatment: Drugs: Placebo for ponsegromab
Double-Blind placebo Treatment followed by Open Label ponsegromab Treatment
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Part A: Change From Baseline in Body Weight at Week 12
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline, Week 12
Query!
Secondary outcome [1]
0
0
Part A: Change From Baseline in Physical Activity at Week 12
Query!
Assessment method [1]
0
0
Physical activity was monitored using accelerometry (wearable digital sensors). Physical activity was categorized as: sedentary activity, non-sedentary physical activity, and moderate to vigorous physical activity. In this outcome measure time for each type of physical activity per day was considered. Baseline was defined as the mean taken over the 8 days of wear during screening. Mean taken over the 8 days of wear before Week 12 was considered. Analysis was performed using mixed models repeated measures (MMRM) model.
Query!
Timepoint [1]
0
0
Baseline, Week 12
Query!
Secondary outcome [2]
0
0
Part A: Change From Baseline in Mean Activity Level During Maximum 6 Minutes at Week 12
Query!
Assessment method [2]
0
0
Physical activity was monitored using accelerometry (wearable digital sensors). In this outcome measure mean activity level during maximum 6 minutes was considered. Baseline was defined as the mean taken over the 8 days of wear during screening. Mean taken over the 8 days of wear before Week 12 was considered. Analysis was performed using MMRM model.
Query!
Timepoint [2]
0
0
Baseline, Week 12
Query!
Secondary outcome [3]
0
0
Part A: Change From Baseline in Total Vector Magnitude at Week 12
Query!
Assessment method [3]
0
0
Total vector magnitude is a measure of overall physical activity. Baseline was defined as the mean taken over the 8 days of wear during screening. Mean taken over the 8 days of wear before Week 12 was considered. Analysis was performed using MMRM model.
Query!
Timepoint [3]
0
0
Baseline, Week 12
Query!
Secondary outcome [4]
0
0
Part A: Change From Baseline in Gait at Week 12
Query!
Assessment method [4]
0
0
Gait was monitored using accelerometry (wearable digital sensors). Analysis was performed using MMRM model. Gait included: gait speed and 95th percentile of gait speed. Baseline was defined as the mean taken over the 8 days of wear during screening. Mean taken over the 8 days of wear before Week 12 was considered. Analysis was performed using MMRM model.
Query!
Timepoint [4]
0
0
Baseline, Week 12
Query!
Secondary outcome [5]
0
0
Part A: Change From Baseline in Functional Assessment of Anorexia-Cachexia Therapy- Anorexia and Cachexia Subscale (FAACT-ACS) at Week 12
Query!
Assessment method [5]
0
0
FAACT-ACS is a 12-item symptom-specific subscale to measure participants' concerns about their anorexia (appetite) or cachexia (weight) for past 7 days. Each item was scored from 0 to 4, where 0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, and 4= very much. The total FAACT-ACS score ranged from 0 to 48. Higher scores are associated with a higher health-related quality of life. FAACT-ACS was analyzed using an MMRM model.
Query!
Timepoint [5]
0
0
Baseline (prior to dose on Day 1), Week 12
Query!
Secondary outcome [6]
0
0
Part A: Change From Baseline in FAACT- 5-Item Anorexia Symptom Scale (5IASS) at Week 12
Query!
Assessment method [6]
0
0
FAACT-5IASS is a 5-item subscale to measure participants' perceptions of anorexia (appetite) concerns for past 7 days. Each item was scored from 0 to 4, where 0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, and 4= very much. The total FAACT-5IASS score ranged from 0 to 20. Higher scores are associated with a higher health-related quality of life. FAACT-5IASS was analyzed using an MMRM model.
Query!
Timepoint [6]
0
0
Baseline (prior to dose on Day 1), Week 12
Query!
Secondary outcome [7]
0
0
Part A: Change From Baseline in Cancer-Related Cachexia Symptom Diary (CRCSD) Scores at Week 12: Appetite, Nausea and Physical Fatigue
Query!
Assessment method [7]
0
0
The CRCSD is a daily, self-reported questionnaire that measured severity of symptoms related to cancer cachexia: appetite, nausea, vomiting, and fatigue. Participants rated appetite, nausea and physical fatigue symptom every day, and weekly averages were calculated over the 7 days prior, from 0 to 10, where 0 = no symptom and 10 = worst possible symptom. Higher scores indicated more severe disease. CRCSD was analyzed using an MMRM model.
Query!
Timepoint [7]
0
0
Baseline, Week 12
Query!
Secondary outcome [8]
0
0
Part A: Median Change From Baseline in CRCSD Scores at Week 12: Vomiting Frequency
Query!
Assessment method [8]
0
0
The CRCSD is a daily, self-reported questionnaire that measured severity of symptoms related to cancer cachexia: vomiting frequency. Participants rated vomiting frequency over the past 24 hours, from 0 to 30, where 0 = no symptom and 30 = worst possible symptom. Higher scores indicated more severe disease.
Query!
Timepoint [8]
0
0
Baseline, Week 12
Query!
Secondary outcome [9]
0
0
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAE)
Query!
Assessment method [9]
0
0
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product, whether or not thought to be related to the investigational product. TEAE were defined as any event that was not present before exposure to study drug, or any event already present that worsened in either intensity or frequency after exposure to study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included serious AEs and all non-SAEs.
Query!
Timepoint [9]
0
0
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)
Query!
Secondary outcome [10]
0
0
Part A: Number of Participants With Incidence of Laboratory Test Abnormalities
Query!
Assessment method [10]
0
0
Laboratory test abnormality parameters included: hematology- hemoglobin (gram per deciliter \[g/dL\]), hematocrit (%), erythrocytes (10\^12/Liter \[L\]) less than (\<) 0.8\*lower limit of normal (LLN); platelets (10\^9/L) \<0.5\*LLN to more than (\>) 1.75\*upper limit of normal (ULN); leukocytes (10\^9/L) \<0.6\*LLN to \>1.5\*ULN; lymphocytes, neutrophils (10\^9/L) \<0.8\*LLN to \>1.2\*ULN. Clinical chemistry- bilirubin, glucose (mg/dL) \>1.5\*ULN; aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase (Units/L \[U/L\]) \>3.0\*ULN; protein, albumin (gram \[g\]/dL) \<0.8\*LLN; urea (mmol/L) \>1.3xULN; creatinine (mg/dL) \>1.3\*ULN; sodium (milliequivalents \[mEq\]/L) \<0.95\*LLN; potassium (mEq/L) \<0.9\*LLN to \>1.1\*ULN.
Query!
Timepoint [10]
0
0
Day 1 up to Week 12
Query!
Secondary outcome [11]
0
0
Part A: Number of Participants With Post-Baseline Vital Signs Meeting the Predefined Criteria
Query!
Assessment method [11]
0
0
Vital signs criteria included: supine systolic blood pressure (SBP) \<90 millimeters of mercury (mmHg), increase and decrease in change of more than or equal to (\>=) 30mmHg; supine diastolic blood pressure (DBP) \<50 mmHg, increase and decrease in change of \>= 20mmHg; pulse rate \<40 beats per minute (bpm) to \>120 bpm. Only rows which included at least 1 participant in any reporting group with abnormality were reported in this outcome measure.
Query!
Timepoint [11]
0
0
Day 1 up to Week 12
Query!
Secondary outcome [12]
0
0
Part A: Number of Participants With Clinically Significant Echocardiogram (ECG) Abnormalities
Query!
Assessment method [12]
0
0
ECG parameters included heart rate (HR), PR interval, QT interval, QTc corrected using Fridericia's formula (QTcF) and QRS complex. HR: RR (interval between 2 successive R waves on ECG) decrease \>25% and to a VR (interval between QRS wave and T wave on ECG) \>100, RR increase \>25% and to a VR \<50; PR interval: baseline less than or equal to (\<=) 200 and % change \>= 50%; QT interval: \>450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; QTcF: 470 \< value \<= 480, 480 \< value \<= 500, value \> 500, 30 \< change \<= 60 and change \>60; QRS complex: value \>= 140, % change \>=50%. Clinically significant values were determined by the investigator.
Query!
Timepoint [12]
0
0
Day 1 up to Week 12
Query!
Eligibility
Key inclusion criteria
Key
* 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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Other
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
21/11/2022
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
23/04/2025
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
187
Query!
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Query!
Recruitment hospital [1]
0
0
St Vincent's Hospital Sydney - Darlinghurst
Query!
Recruitment hospital [2]
0
0
Orange Hospital - Orange
Query!
Recruitment hospital [3]
0
0
Peter MacCallum Cancer Centre - Melbourne
Query!
Recruitment hospital [4]
0
0
Western Health-Sunshine & Footscray Hospitals - St Albans
Query!
Recruitment postcode(s) [1]
0
0
2010 - Darlinghurst
Query!
Recruitment postcode(s) [2]
0
0
2800 - Orange
Query!
Recruitment postcode(s) [3]
0
0
3000 - Melbourne
Query!
Recruitment postcode(s) [4]
0
0
3021 - St Albans
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arkansas
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Indiana
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Louisiana
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Montana
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Oregon
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Texas
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Washington
Query!
Country [9]
0
0
Bulgaria
Query!
State/province [9]
0
0
Burgas
Query!
Country [10]
0
0
Bulgaria
Query!
State/province [10]
0
0
Haskovo
Query!
Country [11]
0
0
Bulgaria
Query!
State/province [11]
0
0
Ruse
Query!
Country [12]
0
0
Bulgaria
Query!
State/province [12]
0
0
Shumen
Query!
Country [13]
0
0
Bulgaria
Query!
State/province [13]
0
0
Sofia
Query!
Country [14]
0
0
Bulgaria
Query!
State/province [14]
0
0
Vratsa
Query!
Country [15]
0
0
Canada
Query!
State/province [15]
0
0
Ontario
Query!
Country [16]
0
0
Canada
Query!
State/province [16]
0
0
Quebec
Query!
Country [17]
0
0
China
Query!
State/province [17]
0
0
Beijing
Query!
Country [18]
0
0
China
Query!
State/province [18]
0
0
Henan
Query!
Country [19]
0
0
China
Query!
State/province [19]
0
0
Hubei
Query!
Country [20]
0
0
China
Query!
State/province [20]
0
0
Jiangsu
Query!
Country [21]
0
0
China
Query!
State/province [21]
0
0
Jiangxi
Query!
Country [22]
0
0
China
Query!
State/province [22]
0
0
Shandong
Query!
Country [23]
0
0
China
Query!
State/province [23]
0
0
Shanghai
Query!
Country [24]
0
0
China
Query!
State/province [24]
0
0
Shanxi
Query!
Country [25]
0
0
China
Query!
State/province [25]
0
0
Zhejiang
Query!
Country [26]
0
0
Hungary
Query!
State/province [26]
0
0
Bács-kiskun
Query!
Country [27]
0
0
Hungary
Query!
State/province [27]
0
0
Jász-nagykun-szolnok
Query!
Country [28]
0
0
Hungary
Query!
State/province [28]
0
0
Budapest
Query!
Country [29]
0
0
Japan
Query!
State/province [29]
0
0
Chiba
Query!
Country [30]
0
0
Japan
Query!
State/province [30]
0
0
Ehime
Query!
Country [31]
0
0
Japan
Query!
State/province [31]
0
0
Hyogo
Query!
Country [32]
0
0
Japan
Query!
State/province [32]
0
0
Kanagawa
Query!
Country [33]
0
0
Japan
Query!
State/province [33]
0
0
Nagoya, Aichi
Query!
Country [34]
0
0
Japan
Query!
State/province [34]
0
0
Shizuoka
Query!
Country [35]
0
0
Japan
Query!
State/province [35]
0
0
Kyoto
Query!
Country [36]
0
0
Japan
Query!
State/province [36]
0
0
Tokyo
Query!
Country [37]
0
0
Poland
Query!
State/province [37]
0
0
Malopolskie
Query!
Country [38]
0
0
Poland
Query!
State/province [38]
0
0
Grudziadz
Query!
Country [39]
0
0
Poland
Query!
State/province [39]
0
0
Katowice
Query!
Country [40]
0
0
Poland
Query!
State/province [40]
0
0
Lublin
Query!
Country [41]
0
0
Slovakia
Query!
State/province [41]
0
0
Banska Bystrica
Query!
Country [42]
0
0
Slovakia
Query!
State/province [42]
0
0
Bratislava
Query!
Country [43]
0
0
Slovakia
Query!
State/province [43]
0
0
Nove Zamky
Query!
Country [44]
0
0
Slovakia
Query!
State/province [44]
0
0
Partizanske
Query!
Country [45]
0
0
Slovakia
Query!
State/province [45]
0
0
Trnava
Query!
Country [46]
0
0
Spain
Query!
State/province [46]
0
0
Barcelona [barcelona]
Query!
Country [47]
0
0
Spain
Query!
State/province [47]
0
0
Catalunya [cataluña]
Query!
Country [48]
0
0
Spain
Query!
State/province [48]
0
0
Illes Balears [islas Baleares]
Query!
Country [49]
0
0
Spain
Query!
State/province [49]
0
0
Valenciana, Comunitat
Query!
Country [50]
0
0
Spain
Query!
State/province [50]
0
0
Madrid
Query!
Country [51]
0
0
Spain
Query!
State/province [51]
0
0
València
Query!
Country [52]
0
0
Taiwan
Query!
State/province [52]
0
0
Tainan
Query!
Country [53]
0
0
Taiwan
Query!
State/province [53]
0
0
Kaohsiung
Query!
Country [54]
0
0
Taiwan
Query!
State/province [54]
0
0
Taichung
Query!
Country [55]
0
0
Taiwan
Query!
State/province [55]
0
0
Taoyuan
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Pfizer
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
Study to evaluate the efficacy, safety and tolerability of ponsegromab compared to placebo in patients with cancer, cachexia, and elevated GDF 15.
Query!
Trial website
https://clinicaltrials.gov/study/NCT05546476
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Pfizer CT.gov Call Center
Query!
Address
0
0
Pfizer
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
Query!
What data in particular will be shared?
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Query!
When will data be available (start and end dates)?
Query!
Available to whom?
Query!
Available for what types of analyses?
Query!
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/76/NCT05546476/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/76/NCT05546476/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT05546476
Download to PDF