IGNITE DMD: Phase 1/2 study of SGT-001 Micro-Dystrophin Gene Transfer in people with Duchenne

IGNITE DMD – Microdystrophin Gene Transfer Study in Adolescents and Children with Duchenne

Questions and Answers

What stage is this research?

In November 2017, Solid Biosciences initiated a Phase 1/2 clinical trial for its lead gene transfer candidate, SGT-001. The clinical trial, called IGNITE DMD, is a randomized, controlled, open-labeled, single-ascending dose study to investigate the safety, tolerability and efficacy of SGT-001 in both ambulatory and non-ambulatory male children and adolescents with Duchenne. For more background and information on the status of this clinical trial, please go to www.solidbio.com/media or www.clinicaltrials.gov.

Where is this research being done and who is funding this research?

The only, current active site for this clinical trial is at the University of Florida. The program is funded by Solid Biosciences.

What is the goal or purpose of this research?

The goal of this research is to evaluate the safety and efficacy of SGT-001, an adeno-associated virus (AAV) mediated gene transfer that contains an engineered version of the dystrophin gene (microdystrophin). The microdystrophin gene in SGT-001 has been designed to produce a functional form of dystrophin protein in skeletal and cardiac muscles. This therapeutic approach is being studied for its potential to treat Duchenne patients, regardless of specific dystrophin mutation.

Who is eligible to participate in this trial?

The IGNITE DMD study is designed to enroll approximately 16 to 32 patients with Duchenne who will be randomly assigned to either an active treatment group or a delayed treatment group. Currently, the study is within the ambulant, children’s cohort ages 4-11, once the Data and Safety Monitoring Board (DSMB) review the data, they may permit non-ambulant adolescents, ages 12-17, to participate again. More information about eligibility for the clinical trial can be found at www.clinicaltrials.gov, NCT03368742.

Where is this clinical trial taking place?

The University of Florida in Gainesville, FL.

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