Contact SCGE




Gene Therapy Trial Report

Summary

DFT383 in Pediatric Participants With Nephropathic Cystinosis


NCTID NCT06910813 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Nephropathic Cystinosis
Disease Ontology Term DOID:1064
Compound Name DFT383
Compound Alias CTNS-RD-04, AVR-RD-04
Sponsor Novartis Pharmaceuticals
Funder Type Industry
Recruitment Status
Enrollment Count 30 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant CTNS
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 Min dose: 3E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-03-17
Completion Date 2044-03-14
Last Update 2025-11-10

Participation Criteria


Eligible Age 2 Years - 5 Years
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Clinical results published 2/18/26

Resources/Links