A Phase I/II clinical trial of the investigational gene therapy SGT-001 designed to treat Duchenne muscular dystrophy has had its clinical hold lifted by the US Food and Drug Administration (FDA), reports Solid Biosciences Inc. The full article can be read here, on the website for Solid Biosciences.
The Phase I/II trial, called Ignite DMD, was placed on clinical hold following a serious adverse event after the first patient received a dose of SGT-001. The patient experienced a reduction in their platelet count and red blood cell count, as well as transient renal impairment, and complement activation (the complement system is a part of the immune system). The patient was treated with steroids and the medication eculizumab. Solid Biosciences reports that he was clinically stable and largely asymptomatic, and the situation is now resolved.
As part of being placed on clinical hold, Solid Biosciences was required to answer questions about the situation from the FDA. The company has now received news from the FDA that the questions were answered satisfactorily and that the clinical hold is lifted. Following this news, Solid is planning to re-start patient enrolment to the study as soon as possible.
The clinical trial has been modified in several ways. These include providing IV glucocorticoids as part of care following dosage of SGT-001, additions to the monitoring protocol, and eculizumab as a treatment option for patients if they experience complement activation.
The experimental gene therapy, SGT-001, is hypothesised to be a treatment for Duchenne muscular dystrophy (DMD) that addresses the underlying cause of the condition. Alterations to the dystrophin gene lead to a lack of functional dystrophin in people with the condition. SGT-001 works by providing patients with a synthetic and functional version of the gene (called microdystrophin), which is thought to then allow the body to produce more functional dystrophin. This is hoped to slow the progression of the condition and is not thought to be dependent on the specific type of gene mutation causing DMD or stage of disease progression. However, more research is needed to establish SGT-001’s effects.
