This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
166
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Take placebo subcutaneously on specified days over a 48 week blinded period
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
Time frame: Baseline
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time frame: Baseline, Week 48
Baseline Time for 4 Stair Climb
The time to complete the 4 stair climb was measured at baseline.
Time frame: Baseline
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Neuromuscular Research Center
Phoenix, Arizona, United States
Stanford University
Palo Alto, California, United States
University of California Davis Medical Center
Sacramento, California, United States
Yale University School of Medicine ; Pulmonary & Critical Care
New Haven, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Rush University Medical Center - PPDS
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
...and 34 more locations
Time frame: Baseline, Week 48
Baseline for the Time to Stand From Supine
The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Time frame: Baseline
Change From Baseline at Week 48 in Stand From Supine Velocity
The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time frame: Baseline, Week 48
Baseline Time for 10 Meter Walk/Run
The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.
Time frame: Baseline
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time frame: Baseline, Week 48
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.
Time frame: Baseline
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time frame: Baseline, Week 48
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.
Time frame: Baseline, Week 48
Baseline for the 6 Minute Walk Distance (6MWD)
The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Time frame: Baseline
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time frame: Baseline, Week 48
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse" at Week 48 as compared to baseline.
Time frame: Baseline, Week 48
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.
Time frame: Baseline, Week 48
Number of Participants With Adverse Events (AEs)
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
Time frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
Number of Participants With AEs Leading to Discontinuation
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.
Time frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)