US-based Solid Biosciences has launched a Phase I/II (IGNITE DMD) clinical trial of its microdystrophin gene transfer candidate SGT-001 in patients with Duchenne muscular dystrophy (DMD).
SGT-001 is an adeno-associated viral (AAV) vector-mediated gene transfer designed to target mutations in the dystrophin gene.
The randomised, controlled, open-label, single-ascending dose Phase I/II trial will assess the safety, tolerability and efficacy of intravenous (IV) dose of SGT-001 in around 16-32 ambulatory and non-ambulatory adolescents and children aged between four and 17 years.
As the trial employs an adoptive model, the firm can adjust the dose and number of subjects over time.
Solid Biosciences founder and CEO Ilan Ganot said: “For more than three years, we’ve been working on a preclinical package and scalable manufacturing process for SGT-001 that would allow us to responsibly move into the clinic.
“This work, as well as decades of research by our scientific advisors and insight from the DMD community, helped us design a clinical trial that will efficiently characterise the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients, regardless of their underlying genetic mutation.”
The trial is being conducted under an investigational new drug (IND) application cleared by the US Food and Drug Administration (FDA) in October.
Various endpoints such as muscle, respiratory and cardiovascular functions, serum and muscle biomarkers related to microdystrophin production and quality of life will be measured during the trial.
Preliminary data from the trial is scheduled to be reported in the first half of 2019.