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

NCT06282432 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 9

Last updated 2025-12-18

No results posted yet for this study

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

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

  • Donald Kohn, MD · University of California, Los Angeles

  • Claire Booth, MBBS, PhD, MSc · University College London Great Ormond Street Institute of Child Health

  • Julian Sevilla, MD, PhD · Hospital Infantil Universitario Niño Jesús

Eligibility

Min Age
3 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-09
Primary Completion
2036-10-04
Completion
2036-10-04

Countries

  • United States
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06282432 on ClinicalTrials.gov