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Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

NCT03157804 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2024-03-21

No results posted yet for this study

Summary

This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .

CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will be inoculated in patients in order to restore their hematopoiesis with genetically corrected stem cells.

Conditions

  • Fanconi Anemia

Interventions

PROCEDURE

IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)

BIOLOGICAL

Genetically Engineered Hematopoietic Stem/Progenitor Cells

Undergo infusion of genetically modified hematopoietic progenitor cell therapy

OTHER

Laboratory Biomarker Analysis

Correlative studies

BIOLOGICAL

Filgrastim

Given subcutaneously (SC)

DRUG

Plerixafor

Given SC

PROCEDURE

Bone Marrow Aspiration

Sponsors & Collaborators

  • Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)

    collaborator UNKNOWN

  • Centro de Investigación en Red de Enfermedades Raras (CIBERER)

    collaborator UNKNOWN

  • Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz

    collaborator OTHER

  • Hospital Vall d'Hebron

    collaborator OTHER

  • Universitat Autonoma de Barcelona

    collaborator OTHER

  • Hospital Infantil Universitario Niño Jesús, Madrid, Spain

    lead OTHER

Principal Investigators

  • Juan A Bueren · CIEMAT/CIBERER/IIS.FJD

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2016-01-07
Primary Completion
2019-04-23
Completion
2023-09-08

Countries

  • Spain

Study Locations

More Related Trials

Entities

Drugs

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 NCT03157804 on ClinicalTrials.gov