News, Research

Solid Biosciences Launches DMD Gene Therapy Trial

Amy Madsen

Solid Biosciences has announced the launch of its first clinical trial for SGT-001, the company’s experimental gene transfer therapy for Duchenne muscular dystrophy (DMD).

The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD.

Enrollment at the first trial site in the United Sates will begin in the coming days, and additional sites are expected to open soon.

Delivering a synthetic dystrophin gene

The underlying cause of DMD is an absence or deficiency of the muscle protein dystrophin, due to a mutation in the DMD gene. SGT-001 is a gene therapy designed to address the genetic cause of the disease via the delivery of highly miniaturized “microdystrophin” replacement genes that enable production of a functional protein to substitute for the missing dystrophin.

Encased in an adeno-associated virus (AAV) vector, or delivery vehicle, microdystrophin genes are administered systemically to the body via intravenous (IV) infusion.

Data from Solid Biosciences’ preclinical program suggests that SGT-001 potentially may be able to slow or stop the progression of DMD, regardless of genetic mutation or stage of disease. The drug has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and European Union.

IGNITE DMD

Goals of the IGNITE DMD trial are to assess the safety and efficacy of a single IV dose of SGT-001 in ambulatory (able to walk) and non-ambulatory boys with DMD, regardless of their underlying genetic mutation.

Investigators will evaluate efficacy based on measurements of the activity, or “expression,” of the microdystrophin gene. They will also assess muscle function and mass, respiratory and cardiovascular function, serum and muscle biomarkers associated with microdystrophin production, patient reported outcomes and quality of life measures.

Trial participants will be randomly assigned to either an active treatment group or a delayed treatment control group. Those in the control group who continue to meet the study’s treatment criteria will receive active treatment after 12 months. All participants will receive a systemic dose of SGT-001.

The study is adaptive, which means the dose and number of participants may be adjusted as the study progresses in order to allow investigators to efficiently and effectively assess the safety and efficacy of SGT-001.

Solid Biosciences has planned to conduct an interim analysis of preliminary data, with results to be announced in the first half of 2019.

Eligibility requirements for participation

Solid Biosciences expects to enroll approximately 16 to 32 participants in the study. Initial details on eligibility criteria include:

  • Participants must be male, with a diagnosis of DMD, between the ages of 4 and 17 years.
  • There is no restriction based on the individual’s underlying gene mutation.
  • All participants must have stable cardiac and respiratory function.
  • All participants must be below a pre-determined threshold for antibodies against AAV; this will be tested as part of the screening process.

Screening and selection will be conducted by investigators at the clinical trial sites.

Find out more

Read Solid Biosciences’ open letter to the DMD community.

More information on IGNITE DMD will be posted on www.clinicaltrials.gov in the coming days. Be sure to check back at mda.org for updates.