NCT03897361View on ClinicalTrials.gov

Stem Cell Gene Therapy for Cystinosis

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

July 8, 2019

Primary Completion Date

September 18, 2024

Study Completion Date

September 18, 2024

Conditions
Lysosomal Storage DiseasesCystinosis
Interventions
GENETIC

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

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.

Trial Locations (1)

92093

University of California San Diego, La Jolla

Sponsors
All Listed Sponsors
collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

collaborator

Cystinosis Research Foundation

OTHER

lead

University of California, San Diego

OTHER