DFT383 in Pediatric Participants With Nephropathic Cystinosis

Conditions

• Nephropathic Cystinosis

Eligibility Criteria

Sex

ALL

Age

2 Years - 5 Years

Healthy Volunteers

Not accepted

Interventions

• DFT383 — GENETIC
  DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.

Study Details

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.

Key Dates

Start date

Jun 2, 2025

Status verified

May 2026

Primary completion

Mar 14, 2031

Completion

Mar 14, 2044

Study Design

Enrollment

30 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1 (DFT383)
  Treatment with DFT383
• No Intervention: Cohort 0 (SoC)
  No study treatment, will continue with standard of care (cysteamine).

Primary Outcome Measure

Core Phase - Incidence of adverse events (Cohort 1) [ Time Frame: Up to 32 months ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals

Locations (4)

Find similar trials in San Diego, CA

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