Status:
RECRUITING
DFT383 in Pediatric Participants With Nephropathic Cystinosis
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Nephropathic Cystinosis
Eligibility:
All Genders
2-5 years
Phase:
PHASE1
PHASE2
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....
Detailed Description
This study is an open-label, multi-center, phase I/II study to assess the safety, tolerability, and efficacy of DFT383 in participants aged 2 to 5 years with nephropathic cystinosis, followed by a lon...
Eligibility Criteria
Inclusion
- 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:
- 1\. Indomethacin within 2 weeks prior to Screening
- Other protocol-defined inclusion/exclusion criteria may apply.
Exclusion
Key Trial Info
Start Date :
June 2 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 14 2044
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT06910813
Start Date
June 2 2025
End Date
March 14 2044
Last Update
November 7 2025
Active Locations (4)
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1
University of California at San Diego - Rady Children's Hospital
San Diego, California, United States, 92123
2
Stanford University - Stanford Children's Health
Stanford, California, United States, 94305
3
Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)
Atlanta, Georgia, United States, 30322
4
Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)
Houston, Texas, United States, 77030