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RecruitingPhase 1/Phase 2

DFT383 in Pediatric Participants With Nephropathic Cystinosis

Novartis Pharmaceuticals · NCT06910813

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Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 2–5 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
30
US sites recruiting
6
Study start
June 2, 2025
Record last updated
October 16, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: DFT383

Conditions: Nephropathic Cystinosis

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Key Inclusion Criteria

Participants eligible for inclusion in this study must meet all the following criteria:

  • Informed consent in writing from parent(s) or legal guardian(s) must be provided
  • 2 to 5 years of age (including 5 years and 364 days old) at Screening
  • Weight-for-stature is ≥ the third percentile, and is ≥ 10 kg
  • Oral cysteamine therapy for at least 6 months
  • Historic clinical diagnosis of nephropathic cystinosis
  • Laboratory evidence of of renal fanconi syndrome (RFS)
  • Relatively preserved kidney function (eGFR ≥ 60mL/min/1.73m2)
  • Received all age-appropriate vaccinations

Key exclusion Criteria for Cohort 1 and 0

  • A history of kidney transplantation
  • A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy
  • History of malignancy
  • A severe or uncontrolled medical disorder
  • 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:

  • Indomethacin within 2 weeks prior to Screening

Other protocol-defined inclusion/exclusion criteria may apply.

Recruiting US locations

  • Phoenix Children's Hospital (Recruiting Cohort 0 and 1)

    Phoenix, AZ 85016

    Site contact: Erica Sieber · 602-933-0171 · esieber@phoenixchildrens.com

  • University of California at San Diego - Rady Children's Hospital (Recruiting Cohort 0 and 1)

    San Diego, CA 92123

    Site contact: Mieko Pretlow · 858-966-1700 · mpretlow@rchsd.org

  • Stanford University - Stanford Children's Health (Recruiting Cohort 0 and 1)

    Stanford, CA 94305

    Site contact: Research Nurse · (650) 725-9032 · scgt_clinical_trials_office@lists.stanford.edu

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

    Atlanta, GA 30322

    Site contact: Laurence (Larry) Greenbaum · 404-712-6374 · lgreen6@emory.edu

  • Children's Hospital of Philadelphia (Recruiting Cohort 0)

    Philadelphia, PA 19104

    Site contact: Melissa Cadnapaphornchai · 215-590-2449

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

    Houston, TX 77030

    Site contact: Ewa Elenberg · 832-824-3800 · Email_elenberg@bcm.edu

Study contacts (from the official record)

  • Novartis Pharmaceuticals · 1-888-669-6682 · novartis.email@novartis.com
  • Novartis Pharmaceuticals
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DFT383 in Pediatric Participants With Nephropathic Cystinosis (NCT06910813) | Bored Studies