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
Eligibility
Sex
ALL
Min age
3 Months
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Enrolled in the Phase I/II Study RP-L201-0318.
2. Received an autologous infusion of CD34+ hematopoietic stem cells modified with a lentiviral vector containing the ITGB2 gene, encoding for the human CD18 receptor in the parent Study RP-L201-0318.
3. Able to adhere to the study visit schedule and other protocol requirements.
4. Provided written informed consent and, as applicable, assent to participate in the current study.
Exclusion Criteria:
There are no criteria for exclusion in this study.
Primary outcome measure(s)
Hematopoietic stem cell transplant (HSCT) free survival — 15 years Survival without allogeneic-HSCT.
Trial sites (3)
Facility
City
Region
Status
University of California, Los Angeles (UCLA)
Los Angeles
California
Hospital Infantil Universitario Niño Jesús
Madrid
Spain
University College London Great Ormond Street Institute of Child Health (GOSH)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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