DFT383 in Pediatric Participants With Nephropathic Cystinosis (NCT06910813) | Clinical Trial Compass
RecruitingPhase 1/2
DFT383 in Pediatric Participants With Nephropathic Cystinosis
United States30 participantsStarted 2025-06-02
Plain-language 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.
Who can participate
Age range
2 Years – 5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Informed consent in writing from parent(s) or legal guardian(s) must be provided
. 2 to 5 years of age (including 5 years and 364 days old) at Screening
. Weight-for-stature is ≥ the third percentile, and is ≥ 10 kg
. Oral cysteamine therapy for at least 6 months
. Historic clinical diagnosis of nephropathic cystinosis
. Laboratory evidence of of renal fanconi syndrome (RFS)
. Relatively preserved kidney function (eGFR ≥ 60mL/min/1.73m2)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Core Phase - Incidence of adverse events (Cohort 1)
Timeframe: Up to 32 months
2
Core Phase - Number of participants with hematological reconstitution (Cohort 1)
Timeframe: 42 days post DFT infusion
3
Core Phase - Proportion of participants with reversal of renal Fanconi syndrome (RFS)