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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04248439 | Gene Therapy for Fanconi Anemia, Complementation Group A | PHASE2 | ACTIVE NOT_RECRUITING | 5 | — | — | Jul 15, 2020 | May 5, 2026 | Dec 22, 2025 | 2 | United States |
Bone Marrow (BM) colony-forming cell (CFC) mitomycin-C (MMC) resistance ≥20% at 12 months post-infusion with a confirmatory result at 18 or 21 months (MMC at 10 nM concentration)
| Arm | Type | Description |
|---|---|---|
| RP-L102 | EXPERIMENTAL | RP-L102 is CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene |
| Name | Type | Description |
|---|---|---|
| RP-L102 | BIOLOGICAL | CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene |
Inclusion Criteria: 1. Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent 2. Subject of the complementation group FA-A 3. Minimum age: 1 year and a minimum weight of 8 kg 4. At least 30 CD34+ cells/μL are det...