Duchenne Muscular Dystrophy
Conditions
Interventions
Genetic: PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Other: Placebo
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
Ot
Sponsors
Kawai Norisuke
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Key inclusion criteria: 1.Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing 2.Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening 3.Ambulatory, as assessed by protocol-specified criteria
Exclusion criteria
Exclusion criteria: Key exclusion criteria: 1.Positive test performed by Pfizer for neutralizing antibodies to AAV9 2.Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna, EXONDYS 51, VYONDYS 53) 3.Any prior treatment with gene therapy 4.Any non-healed injury that may impact functional testing (eg NSAA) 5.Abnormality in specified laboratory tests, including blood counts, liver and kidney function
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Outcome Measures: 1.Change from Baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Week 52 ] The NSAA is a 17-item test that measures gross motor function in children with Duchenne. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Outcome Measures: 1.Change from Baseline in mini-dystrophin expression level in muscle [ Time Frame: Week 52 ] Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry (LC-MS). 2.Change from Baseline in distribution of mini-dystrophin expression in the muscle [ Time Frame: Week 52 ] Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence. 3.Change from Baseline in serum creatine kinase (CK) [ Time Frame: Week 52 ] Changes in the circulating levels of CK. 4.Number of skills gained based on the individual items of the NSAA. [ Time Frame: Week 52 ] To count the skills that each child gained, based on the individual items of the NSAA. 5.Number of skills improved or maintained based on the individual items of the NSAA [ Time Frame: Week 52 ] To count the skills that each child improved or maintained, based on the individual items of the NSAA. 6.Change from Baseline in the 10-meter run/walk test velocity [ Time Frame: Week 52 ] Velocity is calculated based on the time that it takes to complete the 10-meter run/walk test. 7.Change from Baseline in the rise from floor velocity [ Time Frame: Week 52 ] Velocity is calculated based on the time that it takes to the rise from floor. 8.Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale [ Time Frame: Week 52 ] The PODCI contains a list of questions to assess how each caregiver/child evaluates the child's ability to to walk, stand, and perform activities of daily living. 9.Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrucment (PODCI): Sports and Physical Functioning Core Scale [ Time Frame: Week 52 ] The PODCI contains a list of questions to assess how each caregiver/child evaluates the child's ability to perform recreational activities. | — |
Countries
Australia, Belgium, Canada, France, Germany, Israel, Italy, Japan, Russia, South Korea, Spain, Switzerland, Taiwan, United Kingdom, United States
Contacts
Public ContactClinical Trials Information Desk
Pfizer R&D Japan G.K.
Outcome results
None listed