Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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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

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.
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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

1.  Primary:

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

2.  Primary:

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

3.  Secondary:

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

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. ]

5.  Secondary:

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

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. ]

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. ]

8.  Secondary:

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

9.  Secondary:

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

10.  Secondary:

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

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
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