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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06270316 | Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease | PHASE1 | RECRUITING | 12 | — | — | Jun 5, 2024 | Apr 30, 2031 | Oct 23, 2025 | 8 | United States |
| Arm | Type | Description |
|---|---|---|
| Dose Ranging Cohort 1 | EXPERIMENTAL | - |
| Dose Ranging Cohort 2 | EXPERIMENTAL | - |
| Dose Ranging Cohort 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| AMT-191 | DRUG | A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A. Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma levels in patients with Fabry disease. |
Key Inclusion Criteria: * Male of age ≥ 18 years and ≤50 years * Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: 1. Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR 2. α-galactosidase A (GLA) pathogenic or ...