Solid Biosciences Announces Clinical Hold On SGT-001 Phase I/II Clinical Trial For Duchenne Muscular Dystrophy

Solid Biosciences (NASDAQ:SLDB) today announced it has received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold.

As quoted in the press release:

IGNITE DMD is designed to assess the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents with DMD. The first patient dosed in the clinical trial was a non-ambulatory adolescent who received 5E13 vg/kg of SGT-001 on February 14, 2018. 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 patient showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests. The patient responded well to medical treatment and is currently asymptomatic. All laboratory parameters have either improved or returned to normal, and he is continuing outpatient assessments per protocol.

Click here to read the full press release.