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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06092034 | A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | PHASE2 | RECRUITING | 14 | — | — | Sep 5, 2023 | Apr 1, 2032 | May 4, 2026 | 6 | United States, Germany +2 |
Increase of myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).
| Arm | Type | Description |
|---|---|---|
| RP-A501 | EXPERIMENTAL | One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease. |
| Name | Type | Description |
|---|---|---|
| RP-A501 | GENETIC | RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion. |
Key Inclusion Criteria: 1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene. 2. Male. 3. Age ≥8 years. 4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: 1. Abnormal thickening of Left ventricul...