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A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

NCT03812263 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2023-11-15

No results posted yet for this study

Summary

The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201. The primary objectives of the Phase II portion of the study are evaluation of survival, as determined by the proportion of subjects alive at age 2 (24 months) and at least 1-year post-infusion without allogeneic hematopoietic stem cell transplant (HSCT) and characterization of the safety and toxicity associated with the infusion.

Conditions

  • Leukocyte Adhesion Defect - Type I

Interventions

BIOLOGICAL

RP-L201

CD34+ enriched hematopoietic stem cells from subjects with severe LAD-I transduced ex vivo with lentiviral vector carrying the ITGB2 gene, Chim-CD18-WPRE.

Sponsors & Collaborators

  • California Institute for Regenerative Medicine (CIRM)

    collaborator OTHER

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

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

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

  • Julián Sevilla Navarro, MD, PhD · Hospital Infantil Universitario Niño Jesús

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-08-30
Primary Completion
2023-09-12
Completion
2023-09-12
FDA Drug
Yes

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 NCT03812263 on ClinicalTrials.gov