Gene therapy for young children with nephropathic cystinosis
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
This study is testing a new gene therapy for young children aged 2 to 5 with nephropathic cystinosis to see if it works better than the usual treatment.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 2 Years to 5 Years |
| Sex | All |
| Sponsor | Novartis Industry-sponsored |
| Locations | 4 sites (San Diego, California and 3 other locations) |
| Trial ID | NCT06910813 on ClinicalTrials.gov |
What this trial studies
This clinical study evaluates the safety, tolerability, and efficacy of DFT383, a cellular gene therapy, in pediatric participants aged 2 to 5 years with nephropathic cystinosis. The study consists of two cohorts: Cohort 1, which receives DFT383, and Cohort 0, which receives standard of care. Both cohorts will run in parallel, with approximately 15 participants in each, allowing for a comparison of outcomes. The total duration for participants in Cohort 1 is up to 32 months, while those in Cohort 0 will be followed for up to 24 months.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 2 to 5 years with a clinical diagnosis of nephropathic cystinosis who have been on oral cysteamine therapy for at least 6 months.
Not a fit: Patients with a history of kidney transplantation or those who have previously undergone gene therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve the management and outcomes for children suffering from nephropathic cystinosis.
How similar studies have performed: While gene therapy approaches for lysosomal storage disorders are emerging, this specific application in nephropathic cystinosis is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
San Diego, California and 3 other locations
- University of California at San Diego - Rady Children's Hospital — San Diego, California, United States (Recruiting)
- Stanford University - Stanford Children's Health — Stanford, California, United States (Recruiting)
- Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0) — Atlanta, Georgia, United States (Recruiting)
- Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0) — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: 1-888-669-6682
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.