According to Solid Biosciences, the first patient who was subjected to the treatment was hospitalized several days later after laboratory findings revealed reduction in platelet count.
The patient, who was a non-ambulatory adolescent and administered 5E13 vg/kg of SGT-001, later on had a decrease in red blood cell count while showing signs of his complement system getting activated, said the company.
Solid Biosciences said that the patient did not show any signs or symptoms of coagulopathy and there were no relevant variations from baselines in liver function tests.
It added that the patient is currently asymptomatic having responded well to medical treatment. The company said that all laboratory parameters of the patient have either improved or restored to normal condition.
The company, in a statement, said: “Solid reported the event to the FDA and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse Reaction (SUSAR).
“The FDA informed the company that the clinical hold was due to the event. Solid has halted enrollment and dosing in IGNITE DMD and is awaiting the formal Clinical Hold letter from the FDA to understand the requirements for resuming the clinical trial.”
SGT-001 is an adeno-associated viral (AAV) vector-mediated gene transfer which functions by delivering a synthetic dystrophin gene, called microdystrophin, to the body.
This microdystrophin encodes for a functional protein surrogate that is shown in muscles and stabilizes the required associated proteins like the neuronal nitric oxide synthase (nNOS).
SGT-001 was granted rare pediatric disease designation and orphan drug designations from the FDA.
Based in Cambridge in the US state of Massachusetts, Solid Biosciences is a life science focused on developing therapies for DMD.
Image: A phase I/II trial DMD trial of Solid Biosciences’ SGT-001 has been put on clinical trial. Photo: courtesy of jk1991/FreeDigitalPhotos.net.