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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07218887 | ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy | PHASE1 | RECRUITING | 6 | — | — | Oct 24, 2025 | Jan 27, 2032 | Mar 4, 2026 | 7 | United States, Spain |
| Arm | Type | Description |
|---|---|---|
| ALXN2350 | EXPERIMENTAL | Participants will receive one of three dose levels of ALXN2350 depending on the cohort. |
| Name | Type | Description |
|---|---|---|
| ALXN2350 | DRUG | ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion. |
Inclusion Criteria: * Pathogenic or likely pathogenic mutation in BAG3 * Medical history of diagnosis of DCM * Stable combination of HF SoC medications * Adequate acoustic windows for echocardiography Exclusion Criteria: * Presence of antibodies to AAV9 * Presence of a pathogenic or likely pathog...