CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 6 enrolled
Drug / intervention
CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST)genetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03897361
NCT03897361Phase 2Completed
CompareCT.gov

A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing

University of California, San Diego·interventional·Posted Apr 1, 2019·Updated Jan 14, 2026

In Brief

A Phase 2 clinical trial evaluating CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST) for Lysosomal Storage Diseases and Cystinosis. Completed, enrolled 6 participants across 1 site.

Detailed 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 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLysosomal Storage Diseases, Cystinosis
CountriesUnited States
CollaboratorsCalifornia Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation

Timeline

Phase 2CompletedFinished
2020202120222023202420252026
First PostedApr 1, 2019
Enrollment StartJul 8, 2019
Primary CompletionSep 18, 2024
TodayJul 2, 2026
Enrollment to primary: 5.2 yearsPosted 7.3 years ago

Interventions

CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST)genetic

Peripheral blood autologous CD34+ enriched cell fraction transduced with lentiviral vector, pCCL-CTNS or pCDY.EFS.CTNS.T260I, that contains the human CTNS complementary deoxyribonucleic acid (cDNA) sequence.