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Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
Sponsor: Rocket Pharmaceuticals Inc.
Summary
This Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) is a continuation of a Phase 1/2 clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene
Official title: Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene
Key Details
Gender
All
Age Range
3 Months - Any
Study Type
OBSERVATIONAL
Enrollment
9
Start Date
2022-03-09
Completion Date
2036-10-04
Last Updated
2025-12-18
Healthy Volunteers
No
Conditions
Locations (3)
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
Hospital Infantil Universitario Niño Jesús
Madrid, Spain
University College London Great Ormond Street Institute of Child Health (GOSH)
London, United Kingdom