Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

NCT06282432 | Active Not Recruiting

• 1 other identifier: RP-L201-0121-LTFU
• Study Type: observational
• Target: 9
• Locations: 3 countries
• Sites: 3

Brief 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

Conditions

Leukocyte Adhesion Deficiency

Keywords

LAD-I; LAD

Outcome Measures

Primary Outcomes (1)

• Hematopoietic stem cell transplant (HSCT) free survival

  Survival without allogeneic-HSCT.

  15 years

Secondary Outcomes (14)

• Incidence of hospitalizations

  15 years

• Incidence of significant infections

  15 years

• Resolution of LAD-I-related skin rash

  15 years

• Resolution of LAD-I-related periodontal abnormalities

  15 years

• Event free survival

  15 years

Study Arms (1)

Subjects that received RP-L201 on the RP-L201-0318 Parent Study

Subjects that received RP-L201 on the RP-L201-0318 Parent Study and either completed the study or discontinued early.

Eligibility Criteria

• Age: 3 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects that have been treated with RP-L201 on the RP-L201-0318 study.

You may qualify if:

• Enrolled in the Phase I/II Study RP-L201-0318.
• 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.
• Able to adhere to the study visit schedule and other protocol requirements.
• Provided written informed consent and, as applicable, assent to participate in the current study.

Sponsors & Collaborators

• Rocket Pharmaceuticals Inc.: lead

Study Sites (3)

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095-1489, United States

Location

Hospital Infantil Universitario Niño Jesús

Madrid, 28009, Spain

Location

University College London Great Ormond Street Institute of Child Health (GOSH)

London, WC1N 1EH, United Kingdom

Location

Related Publications (1)

• Booth C, Sevilla J, Almarza E, Kuo CY, Zubicaray J, Terrazas D, O'Toole G, Chitty-Lopez M, Choi G, Nicoletti E, Long-Boyle J, Fernandes A, Chetty K, De Oliveira S, Banuelos C, Xu-Bayford J, Bastone AL, John-Neek P, Jackson C, Moore TB, Gilmour K, Schambach A, Rothe M, Kasbekar S, Rao GR, Patel K, Shah G, Thrasher AJ, Bueren JA, Schwartz JD, Kohn DB. Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1. N Engl J Med. 2025 May 1;392(17):1698-1709. doi: 10.1056/NEJMoa2407376.

  PMID: 40305711 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Human Tissue (including blood and bone marrow)

MeSH Terms

Conditions

Leukocyte adhesion deficiency type 1

Study Officials

• Donald Kohn, MD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

• Claire Booth, MBBS, PhD, MSc

  University College London Great Ormond Street Institute of Child Health

  PRINCIPAL INVESTIGATOR

• Julian Sevilla, MD, PhD

  Hospital Infantil Universitario Niño Jesús

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2023
• First Posted: February 28, 2024
• Study Start: March 9, 2022
• Primary Completion (Estimated): October 4, 2036
• Study Completion (Estimated): October 4, 2036
• Last Updated: December 18, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1); GB (1); US (1)