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Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT01099761
Recruitment Status : Terminated (Based on preliminary safety data.)
First Posted : April 8, 2010
Results First Posted : October 14, 2016
Last Update Posted : April 5, 2017
Sponsor:
Information provided by (Responsible Party):
Acceleron Pharma, Inc.

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Duchenne Muscular Dystrophy
Interventions: Biological: ACE-031 0.5 mg/kg q4wk
Biological: ACE-031 1.0 mg/kg q2wk
Other: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
ACE-031 0.5 mg/kg q4wk ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
ACE-031 1.0 mg/kg q2wk ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
ACE-031 ACE-03 2.5 mg/kg q4wk ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
Placebo Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.

Participant Flow:   Overall Study
    ACE-031 0.5 mg/kg q4wk   ACE-031 1.0 mg/kg q2wk   ACE-031 ACE-03 2.5 mg/kg q4wk   Placebo
STARTED   9   9   0 [1]   6 
COMPLETED   9   9   0   6 
NOT COMPLETED   0   0   0   0 
[1] Study terminated prior to enrolling cohort 3



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
ACE-031 0.5 mg/kg q4wk ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
ACE-031 1.0 mg/kg q2wk ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
ACE-031 ACE-03 2.5 mg/kg q4wk ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
Placebo Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Total Total of all reporting groups

Baseline Measures
   ACE-031 0.5 mg/kg q4wk   ACE-031 1.0 mg/kg q2wk   ACE-031 ACE-03 2.5 mg/kg q4wk   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 		 9   9   0   6   24 
Age 
[Units: Participants]
Count of Participants 		         
<=18 years      9 100.0%      9 100.0%         6 100.0%      24 100.0% 
Between 18 and 65 years      0   0.0%      0   0.0%         0   0.0%      0   0.0% 
>=65 years      0   0.0%      0   0.0%         0   0.0%      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants 		         
Female      0   0.0%      0   0.0%         0   0.0%      0   0.0% 
Male      9 100.0%      9 100.0%         6 100.0%      24 100.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants 		         
Hispanic or Latino      0   0.0%      0   0.0%         0   0.0%      0   0.0% 
Not Hispanic or Latino      9 100.0%      9 100.0%         6 100.0%      24 100.0% 
Unknown or Not Reported      0   0.0%      0   0.0%         0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
 		         
Canada   9   9      6   24 
Able to ambulate 10 meters in < 12 seconds 
[Units: Participants]
 		 9   9      6   24 


  Outcome Measures

1.  Primary:   Number of Subjects With Adverse Reactions.   [ Time Frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later ]
  Show Outcome Measure 1

2.  Primary:   Number of Subjects With Clinical Laboratory Adverse Reactions.   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 2

3.  Secondary:   Percent Change in Total Lean Body Mass by DXA Scan.   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 3

4.  Secondary:   Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 4

5.  Secondary:   Percent Change in Muscle Strength Score by Hand-held Myometry.   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 5

6.  Secondary:   Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 6

7.  Secondary:   Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 7

8.  Secondary:   Change in Pulmonary Function Tests (FVC)   [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  Show Outcome Measure 8

9.  Secondary:   Change in Pulmonary Function Test (MIP)   [ Time Frame: Baseline to End-of-Study Visit. approximately 24 weeks ]
  Show Outcome Measure 9

10.  Secondary:   Change in Pulmonary Function Test (MEP)   [ Time Frame: Baseline to End-of-Stuidy Visit, approximately 24 weeks ]
  Show Outcome Measure 10


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Kenneth Attie
Organization: Acceleron Pharma
phone: 617-649-9200 ext 350
e-mail: kattie@xlrn.com



Responsible Party: Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01099761     History of Changes
Other Study ID Numbers: A031-03
First Submitted: April 2, 2010
First Posted: April 8, 2010
Results First Submitted: May 10, 2016
Results First Posted: October 14, 2016
Last Update Posted: April 5, 2017