The US Food and Drug Administration (FDA) has issued a clinical hold on Solid Biosciences’ Phase I/II IGNITE DMD study of SGT-001 to treat Duchenne muscular dystrophy (DMD).
SGT-001 is an adeno-associated viral (AAV) vector-mediated gene transfer designed to target the underlying genetic cause of DMD.
A total of six patients have been dosed in the IGNITE DMD trial. Three patients in the first cohort received a 5E13 vector genomes per kilogram (vg/kg) dose, while three participants in the second cohort were dosed with 2E14vg/kg.
Solid Biosciences said that five patients were doing well, but one in the 2E14vg/kg cohort had a serious adverse event (SAE), which led to reduced red blood cell count, complement activation, acute kidney injury and cardio-pulmonary insufficiency.
The company noted the absence of abnormalities relating to cytokine and coagulopathy.
After reporting the event to the FDA and the Data Safety Monitoring Board (DSMB), the company received a notification from the regulator to place the trial on hold.
The company said that it would work with the regulator to resolve the hold.
Solid Biosciences CEO, president and co-founder Ilan Ganot said: “We are encouraged that this patient is recovering. In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001.
“We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the programme.”
The clinical hold follows the first one in March last year, when a patient dosed with 5E13vg/kg of SGT-001 experienced a suspected unexpected serious adverse reaction (SUSAR).
Solid Biosciences resumed the trial later in June after making certain changes to the study protocol.