Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Condition(s) studied

Investigational drug(s) / intervention(s)

PF-06939926: PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.

Placebo: Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.

Placebo: Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.

PF-06939926: PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2

Study 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.

Eligibility

Primary outcome measure(s)

• Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 — Baseline, Week 52
  The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. Baseline NSAA total score is defined as the last non-missing NSAA total score collected prior to Year 1 drug administration.

Trial sites (53)

+ 13 more sites — see the full list on the official registry below.

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