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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06910813 | DFT383 in Pediatric Participants With Nephropathic Cystinosis | PHASE1 | RECRUITING | 30 | — | — | Jun 2, 2025 | Mar 14, 2044 | May 29, 2026 | 4 | United States |
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Hematological reconstitution by Day 42 post-DFT383 infusion
Proportion of participants with reversal of renal Fanconi syndrome (RFS)
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (DFT383) | EXPERIMENTAL | Treatment with DFT383 |
| Cohort 0 (SoC) | NO_INTERVENTION | No study treatment, will continue with standard of care (cysteamine). |
| Name | Type | Description |
|---|---|---|
| DFT383 | GENETIC | DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy. |
Key Inclusion Criteria: Participants eligible for inclusion in this study must meet all the following criteria: 1. Informed consent in writing from parent(s) or legal guardian(s) must be provided 2. 2 to 5 years of age (including 5 years and 364 days old) at Screening 3. Weight-for-stature is ≥ th...