{"id":6313,"date":"2018-03-16T14:34:20","date_gmt":"2018-03-16T14:34:20","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=6313"},"modified":"2022-05-03T18:17:34","modified_gmt":"2022-05-03T18:17:34","slug":"microdystrophin-gene-transfer-trial-on-hold","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/microdystrophin-gene-transfer-trial-on-hold\/","title":{"rendered":"Microdystrophin gene transfer trial on Hold"},"content":{"rendered":"

Solid Biosciences announces a clinical hold on SGT-001 microdystrophin gene transfer the clinical Phase I\/II clinical trial for Duchenne muscular dystrophy.\u00a0<\/strong><\/p>\n

Solid Biosciences Inc. announced it had received notification from the\u00a0U.S. Food and Drug Administration\u00a0(FDA) that IGNITE DMD, its Phase I\/II clinical trial for microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold.<\/strong><\/p>\n

What happened?<\/h4>\n

The first patient dosed in the clinical trial was a non-ambulatory adolescent. Several days after administration, the patient was hospitalized\u00a0due to laboratory findings that included a decrease in platelet count, followed by a reduction in red blood cell count and evidence of complement activation.\u00a0*<\/strong>The complement system is an enzyme cascade that helps defend against infection.\u00a0<\/em><\/p>\n

How is the patient now?<\/h4>\n

The patient was admitted to the hospital, received treatment, and he is home with his family with no symptoms.<\/p>\n

What is a clinical hold?<\/h4>\n

Solid reported the event to the\u00a0FDA\u00a0and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse Reaction (SUSAR).<\/p>\n

What\u2019s next?<\/h4>\n

The team at Solid will be working with the principal investigator and FDA to fully understand the cause and nature of this event, as well as identify appropriate next steps as soon as possible.<\/p>\n

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In case you don\u2019t remember the specifics about the microdystrophin and gene therapy we invite you to watch the interview we conducted in London with Dr. Jeffrey Chamberlain PH.D.:\u00a0 Here<\/a><\/p><\/blockquote>\n

 <\/p>\n

About the clinical trial:<\/h4>\n

The Phase I\/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD.\u00a0 IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to characterize the safety and efficacy of SGT-001 efficiently. The patient screening will begin at their first participating study in one location in the United States in the coming days. Solid Biosciences is working to bring on additional sites in the United States and abroad.<\/p>\n

About SGT-001<\/h4>\n

SGT-001 is a novel adeno-associated virus* (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD. SGT-001 is a systemically administered* candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.<\/p>\n

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