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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05629559 | 4D-310 in Adults With Fabry Disease and Cardiac Involvement | PHASE1 | RECRUITING | 18 | — | — | Oct 16, 2022 | Jun 1, 2030 | Feb 21, 2025 | 4 | Australia, Taiwan |
| NCT04519749 | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease | PHASE1 | ACTIVE NOT_RECRUITING | 18 | — | — | Sep 1, 2020 | Jun 1, 2030 | Apr 8, 2024 | 4 | United States |
Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose
| Arm | Type | Description |
|---|---|---|
| 4D-310 Dose Level -1 | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level -1 |
| 4D-310 Dose Level 2 | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level 2 |
| 4D-310 Dose Level 3 | EXPERIMENTAL | Single IV administration of 4D-310 at Dose Level 3 |
| 4D-310 Dose Level 1 (No longer enrolling) | EXPERIMENTAL | No longer enrolling - Single IV administration of 4D-310 at Dose Level 1 |
| 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group A | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level 1 - AAV NAb Titer Group A patients |
| 4D-310 Dose Level 1 - AAV NAb Titer Group B | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level 1 - AAV NAb titer Group B patients |
| 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or B | EXPERIMENTAL | Single IV administration of 4D-310 at Dose Level 2 in AAV NAb titer Group A and/or B patients |
| 4D-310 Dose Expansion | EXPERIMENTAL | Dose expansion cohort of single IV administration of 4D-310 at the selected dose and selected AAV Nab titer group(s) patients |
| Name | Type | Description |
|---|---|---|
| 4D-310 | BIOLOGICAL | Single IV administration of 4D-310 |
Inclusion Criteria: 1. Male or female ≥ 18 to ≤65 years of age 2. Pathogenic GLA mutation consistent with Fabry Disease 3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement 4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT...