Recruiting
Phase 1
Phase 2

DFT383

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06910813

Conditions

Nephropathic Cystinosis

Eligibility Criteria

Sex: All

Age: 2 - 5

Healthy Volunteers: Not accepted

Interventions

DFT383

Study Details

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.

Conditions

Nephropathic Cystinosis

Study ID

NCT06910813

Start date

Jun 2, 2025

Status verified date

Jul, 2026

Completion date

May 28, 2044

Anticipated

Primary completion date

May 28, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 5

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

30 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1 (DFT383)

Treatment with DFT383

no intervention: Cohort 0 (SoC)

No study treatment, will continue with standard of care (cysteamine).

Interventions

DFT383

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

Primary outcome measure

  • Core Phase - Incidence of adverse events (Cohort 1) [ Time Frame: Up to 32 months ]
  • Core Phase - Number of participants with hematological reconstitution (Cohort 1) [ Time Frame: 42 days post DFT infusion ]
  • Core Phase - Proportion of participants with reversal of renal Fanconi syndrome (RFS) [ Time Frame: Up to 32 months ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Locations

University of California at San Diego - Rady Children's Hospital

Recruiting

San Diego, California, United States, 92123

Contacts

Mieko Pretlow

858-966-1700mpretlow@rchsd.org

Principal Investigator:

Nadine Benador nbenador@health.ucsd.edu

Stanford University - Stanford Children's Health

Recruiting

Stanford, California, United States, 94305

Contacts

Research Nurse

(650) 725-9032scgt_clinical_trials_office@lists.stanford.edu

Principal Investigator:

Alice Bertaina

Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Laurence (Larry) Greenbaum

404-712-6374lgreen6@emory.edu

Principal Investigator:

Laurence (Larry) Greenbaum

Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)

Recruiting

Houston, Texas, United States, 77030

Contacts

Ewa Elenberg

832-824-3800Email_elenberg@bcm.edu

Principal Investigator:

Ewa Elenberg

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Jul 31, 2026

Last verified

Jul, 2026

Keywords

  • Cystinosis
  • Nephropathic cystinosis
  • Lysosomal storage disorder
  • CTNS gene
  • DFT383
  • Cellular gene therapy
  • Cysteamine
  • Renal Fanconi syndrome

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-07-31.