C3391003 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Conditions

DUCHENNE MUSCULAR DYSTROPHY (DMD)

Brief summary

Change from Baseline at Week 52 in the NSAA total score.

Detailed description

Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay., Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence., Change from Baseline at Week 52 in serum CK concentration., Number of skills gained at Week 52 based on the individual items of the NSAA., Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA., Change from Baseline at Week 52 in the 10 meter run/walk velocity., Change from Baseline at Week 52 in the rise from floor velocity., Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent)., Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).

Related papers

No related papers found yet.

Interventions

DRUGECULIZUMAB

DRUGFordadistrogene Movaparvovec

DRUGPlacebo for PF-06939926

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change from Baseline at Week 52 in the NSAA total score.	—

Secondary

Measure	Time frame
Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay., Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence., Change from Baseline at Week 52 in serum CK concentration., Number of skills gained at Week 52 based on the individual items of the NSAA., Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA., Change from Baseline at Week 52 in the 10 meter run/walk velocity., Change from Baseline at Week 52 in the rise from floor velocity., Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent)., Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).	—

Measure

Time frame

Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay., Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence., Change from Baseline at Week 52 in serum CK concentration., Number of skills gained at Week 52 based on the individual items of the NSAA., Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA., Change from Baseline at Week 52 in the 10 meter run/walk velocity., Change from Baseline at Week 52 in the rise from floor velocity., Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent)., Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).

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Countries

Belgium, France, Germany, Italy, Spain

Outcome results

None listed