Table 5.
Overview of Systemic AAV Micro-dystrophin Clinical Trials
| Solid Biosciences | Nationwide Children’s Hospital | Pfizer | |
|---|---|---|---|
| ClinicalTrials.gov Identifier | NCT03368742 | NCT03375164 | NCT03362502 |
| Trial name | microdystrophin gene transfer study in adolescents and children with DMD (IGNITE DMD) | systemic gene delivery clinical trials for Duchenne muscular dystrophy | a study to evaluate the safety and tolerability of PF-06939926 gene therapy in Duchenne muscular dystrophy |
| Start date (actual) | December 6, 2017 | December 11, 2017 | January 23, 2018 |
| Completion date (estimated) | March, 2021 | January, 2021 | June 7, 2024 |
| Location | University of Florida | Nationwide Children’s Hospital and Washington University | Duke University, UCLA and University of Utah |
| Responsible party | Solid Biosciences | Jerry R. Mendell | Pfizer |
| Study nature | phase 1 and 2, open-label, randomized, controlled | phase 1 and 2, open-label, non-randomized | phase 1b, open-label, non-randomized |
| Drug name | SGT-001 | rAAVrh74.MHCK7.Micro-dystrophin | PF-06939926 |
| AAV-serotype | AAV-9 | AAV-rh74 | AAV-9 |
| Dose | 3 doses (start at 5 × 1013 vg/kg) | 1 dose (2 × 1014 vg/kg) | 2 doses |
| Patient number | 16 | 12 | up to 12 |
| Patient age | 4 to 17 years | 3 months to 7 years | 5 to 12 years |
| Disease stage | both ambulatory and non-ambulatory | ambulatory only | ambulatory only |
| Corticosteroid use | daily for ≥2 years | no prior use for ≤3 years old; >12 weeks use for ≥4 years old | ≥6 month use and ≥3 month daily use |
| Dystrophin gene mutation | any mutation | Frameshift or nonsense mutation within exons 18-58 | any mutation |
| Pre-Nab to AAV | negative | ≤1:400 | negative |
| Primary outcome | safety and micro-dystrophin expression in biopsy | safety | safety and tolerability |
| Secondary outcome | micro-dystrophin expression in biopsy and motor function | micro-dystrophin expression in biopsy |