DFT383 in Pediatric Participants With Nephropathic Cystinosis
Public ClinicalTrials.gov record NCT06910813. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT06910813
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 30 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years to 5 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 1, 2025
- Primary completion
- May 27, 2031
- Completion
- May 27, 2044
- Last update posted
- Jul 30, 2026
2025 – 2044
United States locations
- U.S. sites
- 5
- U.S. states
- 4
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital (Recruitng Cohort 0 and 1) | Phoenix | Arizona | 85016 | Not yet recruiting |
| University of California at San Diego - Rady Children's Hospital | San Diego | California | 92123 | Recruiting |
| Stanford University - Stanford Children's Health | Stanford | California | 94305 | Recruiting |
| Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0) | Atlanta | Georgia | 30322 | Recruiting |
| Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0) | Houston | Texas | 77030 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06910813, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 30, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06910813 live on ClinicalTrials.gov.