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Completed Phase 1Phase 2 Interventional Results available

Stem Cell Gene Therapy for Cystinosis

NCT03897361
Conditions
Lysosomal Storage Diseases, Cystinosis
Interventions
CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
Genetic
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
La Jolla, California