The U.S. Food and Drug Administration put the phase 1/2 trial of Solid Biosciences Inc.'s SGT-001 drug for treating Duchenne muscular dystrophy on clinical hold after a patient was hospitalized.
Duchenne muscular dystrophy, or DMD, is a genetic disorder characterized by progressive muscle degeneration and weakness.
The Ignite DMD trial was investigating the safety and efficacy of SGT-001, a microdystrophin gene therapy for treating ambulatory and nonambulatory children and adolescents with DMD.
Solid Biosciences said the first patient treated in the study was hospitalized after exhibiting low platelet count, low red blood cell count and signs of complement activation. The patient showed no symptoms of bleeding disorder and no changes in the liver based on liver function tests, Solid Biosciences added.
The FDA classified the reported issue as "suspected unexpected serious adverse reaction," a side effect that is unexpected and not consistent with what is known in the product information.
Solid Biosciences is awaiting FDA's formal clinical hold letter containing the requirements needed to resume the trial. The company has stopped enrolling and dosing patients and will work with the FDA to address the clinical hold.
