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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04105166 | Gene Therapy for Pyruvate Kinase Deficiency (PKD) | PHASE1 | COMPLETED | 4 | — | — | Jul 6, 2020 | Jun 9, 2025 | Sep 5, 2025 | 3 | United States, Spain |
The number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) v.5.0.
| Arm | Type | Description |
|---|---|---|
| RP-L301 | EXPERIMENTAL | RP-L301 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene |
| Name | Type | Description |
|---|---|---|
| RP-L301 | BIOLOGICAL | Autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene |
Inclusion Criteria: * PKD diagnosis with a confirmed PKLR mutation. * Adult Cohort ≥18 years old and \<50 years for the initial 2 patients enrolled; Pediatric Cohort ≥8-17 years for the next 2-3 patients. * History of severe, transfusion-dependent anemia, defined as: 1. At least 6 red blood cell...