FDA Lifts Clinical Hold on Trial for Duchenne Muscular Dystrophy Treatment

SGT-001 is a novel adeno-associated viral (AAV) vector mediated gene transfer
SGT-001 is a novel adeno-associated viral (AAV) vector mediated gene transfer

Solid Biosciences will resume their Phase 1/2 clinical trial for their investigational Duchenne muscular dystrophy (DMD) treatment, SGT-001, after the Food and Drug Administration (FDA) removed a hold on the trial.

SGT-001 is a novel adeno-associated viral (AAV) vector mediated gene transfer that could potentially address the underlying genetic cause of DMD, mutations in the dystrophin gene. The hold was placed on the IGNITE DMD trial after the first patient administered SGT-001 had a serious event characterized by a decrease in platelet count followed by a reduction in red blood cell count, transient renal impairment and evidence of complement activation.

The patient received a modified steroid regimen and a limited course eculizumab for the observed complement activation and the event was fully resolved. The FDA stated that the Company addressed the hold questions satisfactorily.

"Our patient quickly returned to his normal activities and planned study assessments," said Barry Byrne, MD, PhD, University of Florida Powell Gene Therapy Center. "We look forward to continuing the IGNITE DMD study and providing additional children and adolescents with this promising investigational therapy."  

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The trial has been amended to now include IV glucocorticoids in the initial weeks after SGT-001 administration and enhanced monitoring measures will include a panel for complement activation. Eculizumab will also be available as a treatment option if complement activation is observed.

For more information visit solidbio.com.