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Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Sponsor: Rocket Pharmaceuticals Inc.
Summary
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
14
Start Date
2022-02-01
Completion Date
2038-05
Last Updated
2026-04-14
Healthy Volunteers
No
Conditions
Interventions
RP-L102
CD34+ enriched cells from subjects with Fanconi anemia subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the FANCA gene, PGK-FANCA-WPRE
Locations (3)
Lucille Packard Children's Hospital, Stanford University
Palo Alto, California, United States
Hospital Infantil Universitario Niño Jesús
Madrid, Spain
University College London Great Ormond Street Institute of Child Health (GOSH)
London, United Kingdom