Study Stopped
Based on preliminary safety data.
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
1 other identifier
interventional
24
1 country
5
Brief Summary
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies. \[Note: This study was terminated based on safety data\]
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Apr 2010
Shorter than P25 for phase_2
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2010
CompletedFirst Submitted
Initial submission to the registry
April 2, 2010
CompletedFirst Posted
Study publicly available on registry
April 8, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2011
CompletedResults Posted
Study results publicly available
October 14, 2016
CompletedOctober 13, 2022
September 1, 2022
1.2 years
April 2, 2010
May 10, 2016
September 14, 2022
Conditions
Outcome Measures
Primary Outcomes (2)
Number of Subjects With Adverse Reactions.
Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug
From treatment initiation to End-of-Study Visit, approximately 24 weeks later
Number of Subjects With Clinical Laboratory Adverse Reactions.
Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug
Baseline to End-of-Study Visit, approximately 24 weeks later.
Secondary Outcomes (8)
Percent Change in Total Lean Body Mass by DXA Scan.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent Change in Muscle Strength Score by Hand-held Myometry.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).
Baseline to End-of-Study Visit, approximately 24 weeks later.
Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).
Baseline to End-of-Study Visit, approximately 24 weeks later.
- +3 more secondary outcomes
Study Arms (3)
ACE-031 0.5 mg/kg q4wk
EXPERIMENTALACE-031 1.0 mg/kg q2wk
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
Eligibility Criteria
You may qualify if:
- Diagnosis of DMD confirmed
- Ambulant
- Corticosteroid therapy for at least one year prior to study day 1 and on a stable dose and schedule for at least 6 months prior to study day 1
- Evidence of muscle weakness by clinical assessment
You may not qualify if:
- Any previous treatment with another investigational product within 6 months prior to study day 1
- Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease that is not related to DMD
- Inability to perform a whole body dual x-ray absorptiometry (DXA) scan
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
Acceleron Investigative Site
Calgary, Alberta, Canada
Acceleron Investigative Site
Vancouver, British Columbia, Canada
Acceleron Investigative Site
Hamilton, Ontario, Canada
Acceleron Investigative Site
London, Ontario, Canada
Acceleron Investigative Site
Ottawa, Ontario, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Senior Vice President, Global Clinical Development
- Organization
- Merck Sharp & Dohme LLC
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 2, 2010
First Posted
April 8, 2010
Study Start
April 1, 2010
Primary Completion
June 1, 2011
Study Completion
June 1, 2011
Last Updated
October 13, 2022
Results First Posted
October 14, 2016
Record last verified: 2022-09
Data Sharing
- IPD Sharing
- Will not share
Abstract summarizing trial data has been published online in Muscle \& Nerve 23-Dec-2016 https://www.ncbi.nlm.nih.gov/pubmed/27462804 The Sponor currently has no plans to make IPD available for a number of reasons; (i) the study was terminated by the Sponsor prematurely based upon concerns for potential adverse drug reactions following long-term use, which were identified in chronic toxicity studies in animals, (ii) the study population was one with a rare disease, in a groups of subjects with a relatively narrow age range, across a small number of study sites. The Sponsor believes that these factors, taken together, make patient anonymity a significant challenge.