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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05885412 | A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) | PHASE1 | RECRUITING | 9 | — | — | Aug 29, 2023 | Sep 1, 2029 | Apr 16, 2026 | 3 | United States |
Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)
| Arm | Type | Description |
|---|---|---|
| RP-A601 | EXPERIMENTAL | Single ascending dose of RP-A601 in 2 consecutive cohorts |
| Name | Type | Description |
|---|---|---|
| RP-A601 | GENETIC | RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a) |
Key Inclusion Criteria: 1. Male or female ≥18 years at the time of signing the informed consent 2. Capable and willing to provide signed informed consent 3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC) 4. Documentation of a pathogenic or likely pathogenic trunc...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |