NCT03812263View on ClinicalTrials.gov

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

August 30, 2019

Primary Completion Date

September 12, 2023

Study Completion Date

September 12, 2023

Conditions
Leukocyte Adhesion Defect - Type I
Interventions
BIOLOGICAL

RP-L201

CD34+ enriched hematopoietic stem cells from subjects with severe LAD-I transduced ex vivo with lentiviral vector carrying the ITGB2 gene, Chim-CD18-WPRE.

Trial Locations (3)

28009

Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid

90095-1489

University of California, Los Angeles, Los Angeles

Unknown

University College London Great Ormond Street Institute of Child Health, London

All Listed Sponsors
collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

lead

Rocket Pharmaceuticals Inc.

INDUSTRY

NCT03812263 - A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Biotech Hunter | Biotech Hunter