Condition: Nephropathic Cystinosis · Sponsor: Novartis Pharmaceuticals
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.
This description comes directly from the study's public registry record.
Novartis Pharmaceuticals · 1-888-669-6682 · novartis.email@novartis.com
Novartis Pharmaceuticals
Always discuss trial participation with your own doctor first.
| 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 |
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Source record: clinicaltrials.gov/study/NCT06910813