A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy

• Research type

  Research Study

• Full title

  A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males with Duchenne Muscular Dystrophy.

• IRAS ID

  1010251

• Contact name

  Kelsey Genda

• Contact email

  kgenda@solidbio.com

• Sponsor organisation

  Solid Biosciences Inc.

• Clinicaltrials.gov Identifier

  NCT06138639

• Research summary

  SGT-003-101 is a first-in-human (FIH), multicenter, open-label Phase 1/2 study to investigate the safety, tolerability, and efficacy of a single IV dose of SGT-003 in ambulant males 4 to <12 years of age with DMD. At least 43 participants will receive SGT-003. Frequent monitoring will occur in the first 45 days following dosing, and participants will be monitored for safety, tolerability, and efficacy for 5 years post-administration of SGT-003 to further evaluate long-term safety.

• REC name

  North East - York Research Ethics Committee

• REC reference

  24/NE/0169

• Date of REC Opinion

  26 Nov 2024

• REC opinion

  Further Information Favourable Opinion