Actively Recruiting
DFT383 in Pediatric Participants With Nephropathic Cystinosis
Led by Novartis Pharmaceuticals · Updated on 2026-04-27
30
Participants Needed
4
Research Sites
980 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
DFT383 in Pediatric Participants With Nephropathic Cystinosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent from parent(s) or legal guardian(s) is required
- Age between 2 and 5 years (up to 5 years and 364 days) at screening
- Weight-for-stature at or above the 3rd percentile and weighs at least 10 kg
- Receiving oral cysteamine therapy for at least 6 months
- History of clinical diagnosis of nephropathic cystinosis
- Laboratory evidence of renal Fanconi syndrome
- Kidney function relatively preserved with eGFR of 60 mL/min/1.73m2 or higher
- Received all age-appropriate vaccinations
You will not qualify if you...
- History of kidney transplantation
- Prior or planned bone marrow or stem cell transplant or prior gene therapy treatment
- History of malignancy
- Severe or uncontrolled medical disorder
- Major surgery within 90 days
- For Cohort 1 only: use of indomethacin within 2 weeks before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
University of California at San Diego - Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
2
Stanford University - Stanford Children's Health
Stanford, California, United States, 94305
Actively Recruiting
3
Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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