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

Running, not enrolling

Conditions studied: Leukocyte Adhesion Deficiency

In brief

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

Key facts

Study ID

NCT06282432

Run by

Rocket Pharmaceuticals Inc.

People needed

9

Starts

2022-03-09

Expected to finish

2036-10-04

Last updated by the study team

2025-12-18

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

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.

You may not qualify if…

• There are no criteria for exclusion in this study.

Where it is running

• 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

Full record on ClinicalTrials.gov

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