A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

Summary

Participation Requirements

Description

This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with Duchenne muscular dystrophy, aged 6 to <12 years (ambulatory subjects only). Approximately 70 subjects will be randomized at a 1:1 ratio to Arm A (pamrevlumab + s...

This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with Duchenne muscular dystrophy, aged 6 to <12 years (ambulatory subjects only). Approximately 70 subjects will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic deflazacort or equivalent potency of corticosteroids administered orally) or Arm B (placebo+ systemic deflazacort or equivalent potency of corticosteroids administered orally), respectively. Subjects must be fully informed of the potential benefits of approved products and make an informed decision when participating in a clinical trial in which they could be randomized to placebo. Subjects will be randomized in a 1:1 ratio to one of the two study treatment arms; pamrevlumab or placebo. Randomization will be stratified by exon 44 deletion. The main study has three study periods: Screening period: Up to 4 weeks Treatment period: 52 weeks Safety Follow-up period/final assessment: A visit 28 days (+/- 3 Days) and a final safety follow-up phone call 60 days (+ 3 Days) after the last dose Each subject will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Subjects who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab. Subjects who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.

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