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Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

NCT04069533 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2024-05-22

No results posted yet for this study

Summary

This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).

Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

Conditions

  • Fanconi Anemia Complementation Group A

Interventions

BIOLOGICAL

RP-L102

CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with lentiviral vector carrying the FANCA gene

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-11-28
Primary Completion
2025-02-28
Completion
2025-02-28
FDA Drug
Yes

Countries

  • 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 NCT04069533 on ClinicalTrials.gov