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NCT06910813 · ClinicalTrials.gov registry record · Phase 1
DFT383 in Pediatric Participants With Nephropathic Cystinosis
A Phase 1 study of Nephropathic Cystinosis, sponsored by Novartis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 30
- Enrollment target
- 4
- Study locations
NCT06910813: Recruiting Phase 1 study of Nephropathic Cystinosis, sponsored by Novartis Pharmaceuticals.
NCT06910813 is a Phase 1 study of Nephropathic Cystinosis that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 30 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (50% lower). The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06910813, a Phase 1 study of Nephropathic Cystinosis, is actively recruiting participants, sponsored by Novartis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 30 participants
- Enrollment target
- 4
- Study locations
Study Summary
An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase. The purpose of this clinical study is to assess safety, tolerability, and efficacy of DFT383 in participants aged 2 to 5 years with nephropathic cystinosis. The study consists of a Core Phase and a long-term Extension Phase. DFT383 is a cellular gene therapy. This study includes an active arm (Cohort 1) of participants treated with study treatment DFT383 and a concurrent reference arm (Cohort 0). Participants in Cohort 0 will not receive study treatment and will only participate in the Core Phase of the study. The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.
Primary Outcome
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Conditions Studied
Interventions
- GENETIC DFT383
Study Locations (4)
California
- University of California at San Diego - Rady Children's Hospital - San Diego
- Stanford University - Stanford Children's Health - Stanford
Georgia
- Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0) - Atlanta
Texas
- Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0) - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2025-06-02 |
| Est. Completion | 2044-03-14 |
| Phase | Phase 1 |
What NCT06910813 shows while recruiting
NCT06910813 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (50% lower).
The record links to 1 condition, with Nephropathic Cystinosis appearing as the primary indexed condition, and to 1 intervention - of which DFT383 is the first listed.
NCT06910813 reports a single indexed study location in California, Georgia, Texas.
Frequently Asked Questions
What is clinical trial NCT06910813 about?
NCT06910813 is a clinical study titled "DFT383 in Pediatric Participants With Nephropathic Cystinosis". An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase. The purpose of this clinical study is to assess safety, tolerability, and efficacy of DFT383 in pa...
What is the current status of trial NCT06910813?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 30 participants. The study started on 2025-06-02. Estimated completion is 2044-03-14.
What conditions does trial NCT06910813 study?
This clinical trial studies the following conditions: Nephropathic Cystinosis.
What interventions are being tested in trial NCT06910813?
The interventions under investigation include: DFT383 (GENETIC).
Who is sponsoring clinical trial NCT06910813?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06910813 being conducted?
This trial has 4 study locations across California, Georgia, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT06910813's enrollment target sits among peer trials
30 1333rd of 2000 higher than 561 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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