Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DFT383 · 1 trial · 1 indication
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...
DFT383 is an investigational gene therapy being developed for the treatment of nephropathic cystinosis, a rare inherited disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
DFT383 is a gene therapy designed to address the underlying genetic cause of nephropathic cystinosis. The specific molecular target or mechanism of action has not been disclosed in available information.
DFT383 is being developed by Novartis AG, a global healthcare company traded on the New York Stock Exchange under the ticker symbol NVS. The drug is in early-stage clinical development.
DFT383 is in Phase 1 clinical development. It is an investigational drug and has not received regulatory approval. The ongoing trial is recruiting participants in the United States.
DFT383 is being evaluated in a Phase 1 clinical trial with the identifier NCT06910813, titled 'DFT383 in Pediatric Participants With Nephropathic Cystinosis.' The study is recruiting up to 30 participants aged 2 years and older in the United States.