Study Stopped
This study was terminated based on preliminary safety data.
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
1 other identifier
interventional
11
1 country
4
Brief Summary
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. \[Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.\]
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 Oct 2010
Shorter than P25 for phase_2
4 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
October 1, 2010
CompletedFirst Submitted
Initial submission to the registry
November 1, 2010
CompletedFirst Posted
Study publicly available on registry
November 11, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2011
CompletedFebruary 1, 2013
January 1, 2013
7 months
November 1, 2010
January 30, 2013
Conditions
Outcome Measures
Primary Outcomes (2)
Number of patients with adverse events.
From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Secondary Outcomes (6)
Percent change in total lean body mass by DXA scan.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in total body and 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 in time to travel 10 meters (standardized 10-Meter-Walk/Run test).
Baseline to End-of-Study Visit, approximately 24 weeks later.
- +1 more secondary outcomes
Study Arms (3)
ACE-031 (Extension of cohort 1 from core study, A031-03)
EXPERIMENTALACE-031 (Extension of cohort 2 from core study, A031-03)
EXPERIMENTALACE-031 (Extension of cohort 3 from core study, A031-03)
EXPERIMENTALInterventions
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Eligibility Criteria
You may qualify if:
- Completion of participation in Study A031-03 and Investigator approval
- Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03
You may not qualify if:
- Participation in any other therapeutic clinical trial
- Plans to have surgery during the course of the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Acceleron Investigative Site
Calgary, Alberta, 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)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 1, 2010
First Posted
November 11, 2010
Study Start
October 1, 2010
Primary Completion
May 1, 2011
Study Completion
May 1, 2011
Last Updated
February 1, 2013
Record last verified: 2013-01