Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT04281485|
Recruitment Status : Recruiting
First Posted : February 24, 2020
Last Update Posted : October 28, 2022
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Genetic: PF-06939926 Other: Placebo||Phase 3|
The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in Cohort 1 and receive gene therapy at the start of the study; approximately one third will be in Cohort 2 and receive placebo at the start of the study and receive gene therapy after one year, as long as it remains safe to do so. The treatment (PF-06939926 gene therapy or placebo) will be given as an intravenous infusion lasting up to 2 hours.
The study includes boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
The primary outcome of the study will be assessed at 52 weeks. All participants will be followed in the study for 5 years after treatment with gene therapy.
The study medication, all medical tests associated with the study, and the visits to the study sites are free of charge. Participants will also be supported for travel costs associated with study visits.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||99 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Parallel up to the measurement of the primary outcome at Week 52. At the beginning of study Year 2 participants who were originally assigned to placebo will have the opportunity to receive PF-06939926. All participants will be followed for 5 years following treatment with PF-06939926.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||The study will be quadruple blind.|
|Official Title:||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|
|Actual Study Start Date :||November 5, 2020|
|Estimated Primary Completion Date :||January 30, 2024|
|Estimated Study Completion Date :||January 29, 2029|
Approximately two thirds of participants will be randomized to Cohort 1.
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
Approximately one third of participants will be randomized to Cohort 2.
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
- 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.
- 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).
- 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.
- Change from Baseline in serum creatine kinase (CK) [ Time Frame: Week 52 ]Changes in the circulating levels of CK.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04281485
|Contact: Pfizer CT.gov Call Center||1-800-718-1021||ClinicalTrials.gov_Inquiries@pfizer.com|
|Study Director:||Pfizer CT.gov Call Center||Pfizer|