MedTrace Logo

Stem Cell Gene Therapy for Cystinosis

NCT03897361 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2026-08-06

Study results available
· View outcomes & findings →

Summary

This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04, including product manufactured with and without the transduction enhancer LentiBOOST \[CTNS-RD-04-LB\])

Conditions

  • Lysosomal Storage Diseases
  • Cystinosis

Interventions

GENETIC

CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)

Cryopreserved autologous CD34+ enriched hematopoietic stem/progenitor cells collected from mobilized peripheral blood and transduced ex vivo with a self-inactivating lentiviral vector (pCCL-CTNS or pCDY.EFS.CTNS.T260I) encoding the human CTNS complementary deoxyribonucleic acid (cDNA) sequence. In later participants, a transduction enhancer (LentiBOOST) was incorporated into the manufacturing process.

Sponsors & Collaborators

Principal Investigators

  • Stephanie Cherqui, Ph.D. · University of California, San Diego

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-07-08
Primary Completion
2024-09-18
Completion
2024-09-18
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03897361 on ClinicalTrials.gov