SGT-001 in male adolescents and children with DMD
Research type
Research Study
Full title
A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy
IRAS ID
233875
Contact name
Thomas Voit
Contact email
Sponsor organisation
Solid Biosciences, LLC
Eudract number
2017-002213-60
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 4 months, 14 days
Research summary
This is a multi-center, randomized, controlled, open-label, single-ascending dose, first-in-human, phase I/II study to evaluate the safety, tolerability, pharmacodynamics and efficacy of a single intravenous (IV) infusion of SGT-001 in subjects with genotypically-confirmed Duchenne muscular dystrophy (DMD). Participation in the study may last up to 2 years. About 16-32 male children and adolescents with DMD between the ages of 4 and 17 will take part in this study. Participants will be assigned to one of two parts of the treatment period – Active Treatment or Delayed Treatment. Two different doses of SGT-001 will be assessed in this study. Participants will receive SGT-001 once and dependent on the part of the study they are randomized to, will be further monitored for another 1 or 2 years. The dose of SGT-001 given will depend on the treatment period the participant is randomized to and the dose that is being explored.
The study will have 3 parts:
-Screening and Randomization;
-Period 1, an open-label, controlled, active treatment phase in which subjects randomized to the active treatment group will receive a single dose of SGT-001 IV;
-Period 2, a delayed treatment phase in which subjects randomized to the control group will receive a single dose of SGT‑001 IV.
The total number of subjects and cohorts, as well as dose escalation and the initiation of delayed treatment in Period 2, will be determined by the Sponsor utilizing acquired data, including both safety data and, when available, relevant efficacy data and Data Safety Monitoring Board recommendations. Initially at least one pair of adolescents aged 12 to 17 years will be enrolled and randomized (1 active treatment, 1 untreated control). After DSMB review of safety data from the first adolescent subject dosed, if safety and tolerability are considered acceptable for proceeding to dosing in children, subjects aged 4 years to 11 years will also be enrolled.
Safety and tolerability will be monitored on an ongoing basis throughout the study and will initially be monitored in an intermediate care unit or equivalent. Efficacy will be investigated periodically throughout the study by measuring microdystrophin expression in muscle (active treatment group only), and using functional assessments, muscle imaging, and biomarkers.
Long-term follow-up will continue in accordance with local regulatory guidelines.REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/1577
Date of REC Opinion
29 Oct 2018
REC opinion
Unfavourable Opinion