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DFT383 in Pediatric Participants With Nephropathic Cystinosis
NCT06910813 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06910813
The ClinicalTrials.gov registry entry for NCT06910813 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Novartis Pharmaceuticals, which has 792 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06910813 reports 4 study locations spanning 3 distinct geographic areas — top geographies include California, Georgia, Texas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06910813?
The interventions under investigation include: DFT383 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06910813?
This trial is sponsored by Novartis Pharmaceuticals, which has 792 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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