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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
Enrollment 24
Recruitment Details  
Pre-assignment Details  
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk ACE-031 ACE-03 2.5 mg/kg q4wk Placebo
Hide Arm/Group Description 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 subcutaneously once every 2 weeks for 12 weeks. ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks. Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Period Title: Overall Study
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
Arm/Group Title 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
Hide Arm/Group Description 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 subcutaneously once every 2 weeks for 12 weeks. ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks. Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks. Total of all reporting groups
Overall Number of Baseline Participants 9 9 0 6 24
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 0 participants 6 participants 24 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  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 0 participants 6 participants 24 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)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 0 participants 6 participants 24 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  
Measure Type: Number
Unit of measure:  Participants
Canada Number Analyzed 9 participants 9 participants 0 participants 6 participants 24 participants
9 9 6 24
Able to ambulate 10 meters in < 12 seconds  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 0 participants 6 participants 24 participants
9 9 6 24
1.Primary Outcome
Title Number of Subjects With Adverse Reactions.
Hide Description Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug
Time Frame From treatment initiation to End-of-Study Visit, approximately 24 weeks later
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Measure Type: Number
Unit of Measure: Number of subjects
1 6 0
2.Primary Outcome
Title Number of Subjects With Clinical Laboratory Adverse Reactions.
Hide Description Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Measure Type: Number
Unit of Measure: Number of subjects
0 0 0
3.Secondary Outcome
Title Percent Change in Total Lean Body Mass by DXA Scan.
Hide Description [Not Specified]
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Error)
Unit of Measure: percentage change in lean body mass
3.6  (1.3) 4.1  (1.5) 2.6  (1.8)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ACE-031 0.5 mg/kg q4wk
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.023
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection ACE-031 1.0 mg/kg q2wk
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.012
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.435
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
4.Secondary Outcome
Title Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.
Hide Description [Not Specified]
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Error)
Unit of Measure: percentage change from baseline
1.6  (1.8) 4.4  (1.5) 0.3  (4.4)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ACE-031 1.0 mg/kg q2wk
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.039
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
5.Secondary Outcome
Title Percent Change in Muscle Strength Score by Hand-held Myometry.
Hide Description Manual Muscle Testing (MMT) is a procedure to measure the function and strength of individual muscles and muscle groups. Hand-held myometry, using a device known as a dynamometer, is one method used for MMT. The dynamometer is held against the patient's limb by the examiner and the patient is asked to resist the force applied by the examiner. The dynamometer measures the force applied by the patient, providing a quantitative and objective assessment of strength of the particular muscle or muscle group. The effectiveness of a therapeutic intervention on muscle strength, as measured by hand-held myometry, can be assessed by comparing post-treatment to pre-treatment (baseline) measurements.
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Deviation)
Unit of Measure: percentage change from baseline
Elbow extension (kg) 0.3  (3.2) 0.1  (2.3) -0.1  (2.1)
Elbow flexion (kg) -0.4  (3.4) -0.7  (2.6) 0.3  (3.1)
Knee extension (kg) -3.6  (4.4) -3.3  (4.4) -3.7  (5.0)
Knee flexion (kg) -2.2  (3.1) -1.9  (4.1) -1.2  (3.9)
6.Secondary Outcome
Title Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).
Hide Description Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test); stratified by baseline age (<10 years vs. >=10 years)
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Deviation)
Unit of Measure: change (m) in 6MWT from baseline
<10 years of age at baseline 43.8  (61.5) 2.5  (35.6) 5.2  (23.6)
>=10 years of age at baseline 4.5  (37.6) -3.2  (35.5) -47.6  (34.5)
7.Secondary Outcome
Title Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).
Hide Description [Not Specified]
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Deviation)
Unit of Measure: Change (sec) in 10MWT from baseline
0.7  (0.9) 0.7  (0.9) 1.0  (0.8)
8.Secondary Outcome
Title Change in Pulmonary Function Tests (FVC)
Hide Description Forced Vital Capacity (FVC); 1 of 3 separate tests employed to assess pulmonary function in this study
Time Frame Baseline to End-of-Study Visit, approximately 24 weeks later.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Deviation)
Unit of Measure: Liters
0.1  (0.2) 0.1  (0.1) 0.1  (0.2)
9.Secondary Outcome
Title Change in Pulmonary Function Test (MIP)
Hide Description Maximum Inspiratory Pressure (MIP); 2 of 3 separate tests employed to assess pulmonary function in this study
Time Frame Baseline to End-of-Study Visit. approximately 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Deviation)
Unit of Measure: cm H2O
17.2  (17.2) 8.7  (7.6) 8.8  (22.1)
10.Secondary Outcome
Title Change in Pulmonary Function Test (MEP)
Hide Description Maximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study
Time Frame Baseline to End-of-Stuidy Visit, approximately 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk Placebo
Hide Arm/Group Description:
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 subcutaneously once every 2 weeks for 12 weeks.
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
Overall Number of Participants Analyzed 9 9 6
Mean (Standard Deviation)
Unit of Measure: cm H2O
11.9  (12.5) 0.8  (3.9) 5.8  (12.1)
Time Frame 24 weeks (12 weeks treatment period + 12 weeks follow up)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk ACE-031 ACE-03 2.5 mg/kg q4wk Placebo
Hide Arm/Group Description 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 subcutaneously once every 2 weeks for 12 weeks. ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks. Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
All-Cause Mortality
ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk ACE-031 ACE-03 2.5 mg/kg q4wk Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Hide Serious Adverse Events
ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk ACE-031 ACE-03 2.5 mg/kg q4wk Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/9 (0.00%)   0/9 (0.00%)   0/0   0/6 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
ACE-031 0.5 mg/kg q4wk ACE-031 1.0 mg/kg q2wk ACE-031 ACE-03 2.5 mg/kg q4wk Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/9 (33.33%)   6/9 (66.67%)   0/0   3/6 (50.00%) 
General disorders         
headache † 1  1/9 (11.11%)  3/9 (33.33%)  0/0  1/6 (16.67%) 
Skin and subcutaneous tissue disorders         
Injection site erythema † 1  3/9 (33.33%)  6/9 (66.67%)  0/0  3/6 (50.00%) 
Vascular disorders         
epistaxis † 1  1/9 (11.11%)  5/9 (55.56%)  0/0  0/6 (0.00%) 
telangiectasia † 1  0/9 (0.00%)  5/9 (55.56%)  0/0  0/6 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[Not Specified]
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
Layout table for Results Point of Contact information
Name/Title: Dr. Kenneth Attie
Organization: Acceleron Pharma
Phone: 617-649-9200 ext 350
EMail: kattie@xlrn.com
Layout table for additonal information
Responsible Party: Acceleron Pharma Inc.
ClinicalTrials.gov Identifier: NCT01099761    
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