Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02122952 | Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | PHASE1 | COMPLETED | 15 | — | — | May 5, 2014 | Dec 15, 2017 | Sep 15, 2022 | 1 | United States |
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | 6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3) |
| Cohort 2 | EXPERIMENTAL | 2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12) |
| Name | Type | Description |
|---|---|---|
| AVXS-101 | BIOLOGICAL | Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter |
Inclusion Criteria: * Six or nine months of age and younger (depending on cohort) on day of vector infusion with Type 1 SMA as defined by the following features: * Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2. ...