FDA Puts Clinical Hold on Duchenne Trial

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

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.

SGT-001, a novel adeno acid-associated viral (AAV) vector-medicated gene transfer, is Solid’s lead drug candidate. It is being investigated for its ability to address the lack of functional dystrophin, which is the protein necessary for keeping muscle cells intact, and the underlying genetic cause of DMD. Preclinical data of the drug suggests its potential to slow or stop disease progression, genetic mutation or disease stage notwithstanding.

The hospitalized patient was administered 5E13 vg/kkg of SGT-001 on February 14, 2018, and shower no signs or symptoms of bleeding disorder, or coagulopathy, and no relevant changes from baseline to liver function.

The patient reportedly responded positively to medical treatment and is currently asymptomatic. All laboratory parameters have either improved or returned to baseline, and outpatient assessments are being continued per protocol.

All enrollment and dosing in IGNITE DMD has been indefinitely discontinued, and the company is awaiting a formal Clinical Hold letter from the FDA to understand the requirements for resuming the clinical trial.

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