FDA Holds Muscular Dystrophy Trial

The U.S. Food and Drug Administration (FDA) has placed a Clinical Hold on the IGNITE DMD Phase 2/3 trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), Solid Biosciences Inc. has announced.

IGNITE DMD, which is designed to assess the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents with DMD, was stopped after the first patient dosed was hospitalized due to laboratory findings that included a decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation.

Solid reported the incident to the FDA and it was immediately classified as a Suspected Unexpected Serious Adverse Reaction (SUSAR) because it was not premeditated or anticipated.

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