NCT06910813 DFT383 in Pediatric Participants With Nephropathic Cystinosis
| NCT ID | NCT06910813 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Novartis Pharmaceuticals |
| Condition | Nephropathic Cystinosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 30 participants |
| Start Date | 2025-06-02 |
| Primary Completion | 2031-03-14 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 30 participants in total. It began in 2025-06-02 with a primary completion date of 2031-03-14.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Key Inclusion Criteria: Participants eligible for inclusion in this study must meet all the following criteria: 1. Informed consent in writing from parent(s) or legal guardian(s) must be provided 2. 2 to 5 years of age (including 5 years and 364 days old) at Screening 3. Weight-for-stature is ≥ the third percentile, and is ≥ 10 kg 4. Oral cysteamine therapy for at least 6 months 5. Historic clinical diagnosis of nephropathic cystinosis 6. Laboratory evidence of of renal fanconi syndrome (RFS) 7. Relatively preserved kidney function (eGFR ≥ 60mL/min/1.73m2) 8. Received all age-appropriate vaccinations Key exclusion Criteria for Cohort 1 and 0 1. A history of kidney transplantation 2. A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy 3. History of malignancy 4. A severe or uncontrolled medical disorder 5. Major surgery within 90 days Additional Key exclusion criteria for Cohort 1 - The following exclusion criterion applies to Cohort 1 only as it is related to DFT383 treatment: 1\. Indomethacin within 2 weeks prior to Screening Other protocol-defined inclusion/exclusion criteria may apply.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06910813 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 5 Years, studying Nephropathic Cystinosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06910813 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06910813 currently recruiting?
Yes, NCT06910813 is actively recruiting participants. Contact the research team at novartis.email@novartis.com for enrollment information.
Where is the NCT06910813 trial being conducted?
This trial is being conducted at San Diego, United States, Stanford, United States, Atlanta, United States, Houston, United States.
Who is sponsoring the NCT06910813 clinical trial?
NCT06910813 is sponsored by Novartis Pharmaceuticals. The trial plans to enroll 30 participants.