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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
Drug Product Name DFT383
Drug Product Alias CTNS-RD-04, AVR-RD-04
Drug Product Description 🔄 pCCL-EFS-CTNS-WPRE
Sponsor Novartis Pharmaceuticals
Funder Type Industry
Recruitment Status
Enrollment Count 30 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant CTNS
Regulatory Element EFS (promoter)
Therapeutic Modality 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
Gene Delivery System Type Viral transduction
Vector Type 🔄 Lentivirus
Viral Vector Subtype HIV-1(VSV-G)
Dose 1 Min dose: 3E6 CD34+ cells/kg

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2025-03-17
Completion Date 🔄 2044-05-28
Last Update 🔄 2026-10-16

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 🔄 6
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