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Clinical Trials in Spain / NCT06282432
Active, not recruiting Observational

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

NCT06282432 · tracked via the Priya Life Science Spain tracker
Sponsor
Rocket Pharmaceuticals Inc
Phase
Observational
Started
2022-03-09
Last updated
2025-12-18

Condition(s) studied

Leukocyte Adhesion Deficiency

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

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)

FacilityCityRegionStatus
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) London United Kingdom

More Rocket Pharmaceuticals Inc. trials in Spain

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 Enrolling by invitation
Danon Disease Natural History Study Recruiting
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy Active, not recruiting
Official registry record

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.

View NCT06282432 on ClinicalTrials.gov ↗ ← All trials in Spain