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 range2 Years – 5 Years
SexALL
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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)
✓. Received all age-appropriate vaccinations
Exclusion criteria
✕. A history of kidney transplantation
✕. A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy
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)