Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Phase 3
Active, not recruiting
NCT04281485
| Condition | Duchenne Muscular Dystrophy |
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| Phase | Phase 3 |
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| Sponsor | Pfizer |
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| Status | Active, not recruiting |
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| Enrollment | 114 participants |
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| Interventions | PF-06939926, PF-06939926 |
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| Start date | Nov 5, 2020 |
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| Completion date | May 15, 2024 |
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| Results posted | Jun 8, 2025 |
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Summary
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Baseline, Week 52 · Score on a scale
| Cohort 1 | 1.46 Score on a scale |
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| Cohort 2 | 1.37 Score on a scale |
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| p-value | =0.9116 |
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| Least Square Mean Difference | 0.09 (95% CI -1.46–1.64) |
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Open in BENITIONS
View on ClinicalTrials.gov
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