Trial Outcomes & Findings for Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy (NCT NCT01099761)
NCT ID: NCT01099761
Last Updated: 2022-10-13
Results Overview
Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
From treatment initiation to End-of-Study Visit, approximately 24 weeks later
Results posted on
2022-10-13
Participant Flow
Participant milestones
| Measure |
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.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
9
|
9
|
0
|
6
|
|
Overall Study
COMPLETED
|
9
|
9
|
0
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
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
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
—
|
6 Participants
n=4 Participants
|
24 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
—
|
6 Participants
n=4 Participants
|
24 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
—
|
6 Participants
n=4 Participants
|
24 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
Canada
|
9 participants
n=5 Participants
|
9 participants
n=7 Participants
|
—
|
6 participants
n=4 Participants
|
24 participants
n=21 Participants
|
|
Able to ambulate 10 meters in < 12 seconds
|
9 participants
n=5 Participants
|
9 participants
n=7 Participants
|
—
|
6 participants
n=4 Participants
|
24 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: From treatment initiation to End-of-Study Visit, approximately 24 weeks laterNumber of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Number of Subjects With Adverse Reactions.
|
1 Number of subjects
|
6 Number of subjects
|
0 Number of subjects
|
PRIMARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Number of Subjects With Clinical Laboratory Adverse Reactions.
|
0 Number of subjects
|
0 Number of subjects
|
0 Number of subjects
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Percent Change in Total Lean Body Mass by DXA Scan.
|
3.6 percentage change in lean body mass
Standard Error 1.3
|
4.1 percentage change in lean body mass
Standard Error 1.5
|
2.6 percentage change in lean body mass
Standard Error 1.8
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.
|
1.6 percentage change from baseline
Standard Error 1.8
|
4.4 percentage change from baseline
Standard Error 1.5
|
0.3 percentage change from baseline
Standard Error 4.4
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.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.
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Percent Change in Muscle Strength Score by Hand-held Myometry.
Elbow extension (kg)
|
0.3 percentage change from baseline
Standard Deviation 3.2
|
0.1 percentage change from baseline
Standard Deviation 2.3
|
-0.1 percentage change from baseline
Standard Deviation 2.1
|
|
Percent Change in Muscle Strength Score by Hand-held Myometry.
Elbow flexion (kg)
|
-0.4 percentage change from baseline
Standard Deviation 3.4
|
-0.7 percentage change from baseline
Standard Deviation 2.6
|
0.3 percentage change from baseline
Standard Deviation 3.1
|
|
Percent Change in Muscle Strength Score by Hand-held Myometry.
Knee extension (kg)
|
-3.6 percentage change from baseline
Standard Deviation 4.4
|
-3.3 percentage change from baseline
Standard Deviation 4.4
|
-3.7 percentage change from baseline
Standard Deviation 5.0
|
|
Percent Change in Muscle Strength Score by Hand-held Myometry.
Knee flexion (kg)
|
-2.2 percentage change from baseline
Standard Deviation 3.1
|
-1.9 percentage change from baseline
Standard Deviation 4.1
|
-1.2 percentage change from baseline
Standard Deviation 3.9
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test); stratified by baseline age (\<10 years vs. \>=10 years)
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).
<10 years of age at baseline
|
43.8 change (m) in 6MWT from baseline
Standard Deviation 61.5
|
2.5 change (m) in 6MWT from baseline
Standard Deviation 35.6
|
5.2 change (m) in 6MWT from baseline
Standard Deviation 23.6
|
|
Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).
>=10 years of age at baseline
|
4.5 change (m) in 6MWT from baseline
Standard Deviation 37.6
|
-3.2 change (m) in 6MWT from baseline
Standard Deviation 35.5
|
-47.6 change (m) in 6MWT from baseline
Standard Deviation 34.5
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).
|
0.7 Change (sec) in 10MWT from baseline
Standard Deviation 0.9
|
0.7 Change (sec) in 10MWT from baseline
Standard Deviation 0.9
|
1.0 Change (sec) in 10MWT from baseline
Standard Deviation 0.8
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit, approximately 24 weeks later.Forced Vital Capacity (FVC); 1 of 3 separate tests employed to assess pulmonary function in this study
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Change in Pulmonary Function Tests (FVC)
|
0.1 Liters
Standard Deviation 0.2
|
0.1 Liters
Standard Deviation 0.1
|
0.1 Liters
Standard Deviation 0.2
|
SECONDARY outcome
Timeframe: Baseline to End-of-Study Visit. approximately 24 weeksMaximum Inspiratory Pressure (MIP); 2 of 3 separate tests employed to assess pulmonary function in this study
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Change in Pulmonary Function Test (MIP)
|
17.2 cm H2O
Standard Deviation 17.2
|
8.7 cm H2O
Standard Deviation 7.6
|
8.8 cm H2O
Standard Deviation 22.1
|
SECONDARY outcome
Timeframe: Baseline to End-of-Stuidy Visit, approximately 24 weeksMaximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study
Outcome measures
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 Participants
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
n=9 Participants
ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
|
Placebo
n=6 Participants
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|
|
Change in Pulmonary Function Test (MEP)
|
11.9 cm H2O
Standard Deviation 12.5
|
0.8 cm H2O
Standard Deviation 3.9
|
5.8 cm H2O
Standard Deviation 12.1
|
Adverse Events
ACE-031 0.5 mg/kg q4wk
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
ACE-031 1.0 mg/kg q2wk
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
ACE-031 ACE-03 2.5 mg/kg q4wk
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
ACE-031 0.5 mg/kg q4wk
n=9 participants at risk
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
n=9 participants at risk
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
n=6 participants at risk
Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
|
|---|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Injection site erythema
|
33.3%
3/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
66.7%
6/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
—
0/0 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
50.0%
3/6 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
|
Vascular disorders
epistaxis
|
11.1%
1/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
55.6%
5/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
—
0/0 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
0.00%
0/6 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
|
Vascular disorders
telangiectasia
|
0.00%
0/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
55.6%
5/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
—
0/0 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
0.00%
0/6 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
|
General disorders
headache
|
11.1%
1/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
33.3%
3/9 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
—
0/0 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
16.7%
1/6 • 24 weeks (12 weeks treatment period + 12 weeks follow up)
|
Additional Information
Senior Vice President, Global Clinical Development
Merck Sharp & Dohme LLC
Phone: 1-800-672-6372
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place