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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 is a Phase 1 study of Nephropathic Cystinosis that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 30 participants. The trial reports 4 study locations across 3 states.
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.
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 |
Interested in This Trial?
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, categorized as Phase 1. 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 1,375 total studies on file at ClinicalTrials.gov.
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 4 study locations spanning 3 distinct geographic areas - top geographies include 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.
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