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

  t.voit@ucl.ac.uk

• Sponsor organisation

  Solid Biosciences, LLC

• Eudract number

  2017-002213-60

• Clinicaltrials.gov Identifier

  NCT03368742

• Duration of Study in the UK

  7 years, 6 months, days

• Research summary

  This is a multi-center, randomized, controlled, open-label, single-ascending dose, FIH, phase I/II study evaluating the safety, tolerability, pharmacodynamics and efficacy of a single intravenous (IV) infusion of SGT-001 in subjects with genotypically-confirmed Duchenne muscular dystrophy. Participation in the study will be approximately 5 years 2 months. About 16-32 male children and adolescents between the ages of 4 and 17 will be enrolled. Participants will be assigned to either the Active Treatment or Delayed Treatment groups. It is planned that a range of doses will be assessed in the study, with a starting dose of 5E13 vg/kg, with possible escalation to a maximum dose of 2E14 vg.kg. Participants will receive SGT-001 once and will have follow-up for 5 years following dosing. 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 4 parts:
  - Screening and Randomization;
  - Period 1, an open-label, controlled, active treatment phase in which subjects randomized to the active treatment group (ATG) 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.
  - Long-term follow-up of dosed subjects will be conducted yearly for a minimum of 5 years after SGT-001 administration.
  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 safety data and, when available, relevant efficacy data and DSMB recommendations.
  Safety will be monitored on an ongoing basis and will initially be monitored in an intermediate care unit. Efficacy will be investigated by measuring microdystrophin expression in muscle (ATG), and using functional assessments, muscle imaging, and biomarkers.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  19/LO/0163

• Date of REC Opinion

  4 Feb 2019

• REC opinion

  Unfavourable Opinion