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

NCT ID: NCT06282432

Last Updated: 2025-12-18

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 9 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-03-09
• Study Completion Date: 2036-10-04

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

Detailed Description

Following the end of participation in Study RP-L201-0318, patients will be offered enrollment into this LTFU protocol. Patients will be followed for up to 15 years following the RP-L201 infusion in the parent study, until the patient dies, withdraws consent, or is lost to follow-up (whichever occurs first).

For all follow-up visits, remote evaluation facilitated by local health care providers (with blood sample shipment to relevant laboratory facilities) is permitted; however, annual visits to the study center are required during initial 3 years post- RP-L201 infusion. Visits where a bone marrow sample is being collected are required to be performed at the study center for the duration of the study. Peripheral Blood samples and bone marrow samples will be archived and tested when clinically or scientifically indicated, as in the event of development of a second malignancy.

Conditions

Leukocyte Adhesion Deficiency

Keywords

LAD-I; LAD

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Study Groups

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.

No interventions assigned to this group

Eligibility Criteria

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.

• Minimum Eligible Age: 3 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Rocket Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Donald Kohn, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Claire Booth, MBBS, PhD, MSc

Role: PRINCIPAL_INVESTIGATOR

University College London Great Ormond Street Institute of Child Health

Julian Sevilla, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Hospital Infantil Universitario Niño Jesús

Locations

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

Countries

United States; Spain; United Kingdom

References

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.

Reference Type: DERIVED

PMID: 40305711 (View on PubMed)

Other Identifiers

RP-L201-0121-LTFU

Identifier Type: -

Identifier Source: org_study_id