Summary of clinical studies supporting approval of etranacogene dezaparvovec
| Characteristic . | Phase 1 open-label dose escalation AMT-060 . | Phase 2b open-label AMT-061 . | Phase 3 open-label AMT-061 . |
|---|---|---|---|
| Number of participants infused | 10 | 3 | 54 |
| Dose(s) | 5 × 1012 gc/kg 2 × 1013 gc/kg | 2 × 1013 gc/kg | 2 × 1013 gc/kg |
| Ages, mean (range), y | 54 (33-72) | 46.7 (43-50) | 41.5 (19-75) |
| Severity of hemophilia | 9 severe (<1%) 1 moderate (1.5%) | 2 severe (<1%) 1 moderate (1%) | 44 severe 10 moderately severe (≤2%) |
| FIX prophylaxis before study entry | 9 on prophylaxis 1 on demand | 3 on prophylaxis | 54 on prophylaxis 4 on-demand∗ |
| HIV-positive | 1/10 participants | 2/3 participants | 3/54 participants |
| Previous HCV infection | 6/10 participants | 3/3 participants | 28/54 participants |
| Mean FIX activity (1-stage aPTT-based FIX) after vector infusion | Cohort 1, 4.4 IU/dl (95% CI, 1.5-7.3 IU/dl) at 52 wk; Cohort 2, 6.9 IU/dl (95% CI, 2.6-11.3 IU/dl) at 26 wk | 47% normal (33.2%-57%) at 26 wk | Change from baseline: 36.2% at 6 mo 38.8% at 12 mo 34.3% at 18 mo |
| Characteristic . | Phase 1 open-label dose escalation AMT-060 . | Phase 2b open-label AMT-061 . | Phase 3 open-label AMT-061 . |
|---|---|---|---|
| Number of participants infused | 10 | 3 | 54 |
| Dose(s) | 5 × 1012 gc/kg 2 × 1013 gc/kg | 2 × 1013 gc/kg | 2 × 1013 gc/kg |
| Ages, mean (range), y | 54 (33-72) | 46.7 (43-50) | 41.5 (19-75) |
| Severity of hemophilia | 9 severe (<1%) 1 moderate (1.5%) | 2 severe (<1%) 1 moderate (1%) | 44 severe 10 moderately severe (≤2%) |
| FIX prophylaxis before study entry | 9 on prophylaxis 1 on demand | 3 on prophylaxis | 54 on prophylaxis 4 on-demand∗ |
| HIV-positive | 1/10 participants | 2/3 participants | 3/54 participants |
| Previous HCV infection | 6/10 participants | 3/3 participants | 28/54 participants |
| Mean FIX activity (1-stage aPTT-based FIX) after vector infusion | Cohort 1, 4.4 IU/dl (95% CI, 1.5-7.3 IU/dl) at 52 wk; Cohort 2, 6.9 IU/dl (95% CI, 2.6-11.3 IU/dl) at 26 wk | 47% normal (33.2%-57%) at 26 wk | Change from baseline: 36.2% at 6 mo 38.8% at 12 mo 34.3% at 18 mo |
aPTT, activated partial thromboplastin time; CI, confidence interval; HCV, hepatitis C virus.
Some participants received both on-demand and prophylaxis during the lead-in period.