Microdystrophin gene transfer trial on Hold

Solid Biosciences Announces Clinical Hold On SGT-001 microdystrophin gene transfer Clinical Phase I/II Clinical Trial for Duchenne Muscular Dystrophy. 

Solid Biosciences Inc. announced it had received notification from the U.S. Food and Drug Administration (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.

What happened?

The first patient dosed in the clinical trial was a non-ambulatory adolescent. Several days after administration the patient was hospitalized 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. *The complement system is an enzyme cascade that helps defend against infection. 

How is the patient now?

The patient was admitted to the hospital, received treatment, and he is home with his family with no symptoms.

What is a clinical hold?

Solid reported the event to the FDA and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse Reaction (SUSAR).

What’s next?

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.

 

In case you don’t 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.:  Here

 

About the clinical trial:

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.  IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001. 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.

About SGT-001

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.

 

  • Clinical trial: Here
  • Letter to the Duchenne Community About the Status of the IGNITE DMD Clinical Trial: Here
  • Link to press release: Here
  • More info on our previous blog post: Here
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