DFT383 in Pediatric Participants With Nephropathic Cystinosis
NCT ID: NCT06910813
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
30 participants
INTERVENTIONAL
2025-06-02
2044-03-14
Brief Summary
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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.
Detailed Description
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The study includes two Treatment Groups (Cohort 1 and Cohort 0) and consists of a Core Phase and a long-term Extension Phase.
Participants in Cohort 1 will receive DFT383 and participate in both the Core and Extension Phase. Participants in Cohort 0 will not receive study treatment and will participate in the Core Phase only.
The two cohorts will be run in parallel. Investigational sites may participate in one or both cohorts.
Cohort 1 Approximately 15 participants will receive treatment with DFT383 in 3 (sub) cohorts (1A, 1B and 1C) dosed in a staggered approach. The total study duration for a participant in Cohort 1 will be up to 32 months in the core phase and up to 13 years for the long-term extension phase.
Cohort 0 Approximately 15 participants meeting similar inclusion/exclusion criteria and receiving SoC will be enrolled. The Schedule of Activities will be reduced for this Cohort. This cohort 0 is not a direct control but will provide essential context for interpreting the results observed in the participants receiving DFT383. The total study duration for a participant in Cohort 0 will be up to 24 months.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1 (DFT383)
Treatment with DFT383
DFT383
DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.
Cohort 0 (SoC)
No study treatment, will continue with standard of care (cysteamine).
No interventions assigned to this group
Interventions
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DFT383
DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.
Eligibility Criteria
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Inclusion 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
Exclusion Criteria
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
1\. Indomethacin within 2 weeks prior to Screening
2 Years
5 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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University of California at San Diego - Rady Children's Hospital
San Diego, California, United States
Stanford University - Stanford Children's Health
Stanford, California, United States
Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)
Atlanta, Georgia, United States
Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)
Houston, Texas, United States
Countries
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Central Contacts
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Novartis Pharmaceuticals
Role: CONTACT
Facility Contacts
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Caitlin Carter
Role: primary
Research Nurse
Role: primary
Laurence (Larry) Greenbaum
Role: primary
Ewa Elenberg
Role: primary
Other Identifiers
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CDFT383A12101
Identifier Type: -
Identifier Source: org_study_id