The US Food and Drug Administration (FDA) has issued a notification to put Solid Biosciences’ IGNITE DMD clinical on hold.
The clinical hold notice was issued after an unexpected event classified as a Suspected Unexpected Serious Adverse Reaction (SUSAR) was reported in the Phase I/II trial of SGT-001 for the treatment of Duchenne muscular dystrophy (DMD).
IGNITE DMD is intended to evaluate the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents with DMD.
Last month, Solid dosed the trial’s first patient, a non-ambulatory adolescent, with 5E13vg/kg of SGT-001.
Shortly after the administration the patient was hospitalised 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 Biosciences said in a statement: “The patient showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests.
“The patient responded well to medical treatment and is currently asymptomatic.
“All laboratory parameters have either improved or returned to normal, and he is continuing outpatient assessments per protocol.”
The event was reported to FDA, which issued a clinical hold on the IGNITE DMD trial.
Following the hold, Solid has stopped enrolment and dosing in IGNITE DMD and is awaiting receipt of the formal Clinical Hold letter from the FDA to understand the requirements for resuming the trial.
The company’s lead candidate, SGT-001, is a new adeno-associated viral (AAV) vector-mediated gene transfer being investigated for its ability to address the underlying genetic cause of DMD. In the condition mutations in the dystrophin gene result in the absence or near-absence of dystrophin protein.
SGT-001 is a systemically administered solution that provides a synthetic dystrophin gene, known as microdystrophin, to the body.