NCT03157804 - Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

January 7, 2016

Primary Completion Date

April 23, 2019

Study Completion Date

September 8, 2023

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

Trial Locations (2)

28009

Hospital Infantil del Niño Jesus, Madrid

08035

Hospital Vall d'Hebron, Barcelona

All Listed Sponsors

collaborator

Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)

UNKNOWN

collaborator

Centro de Investigación en Red de Enfermedades Raras (CIBERER)

UNKNOWN

collaborator

Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz

OTHER

collaborator

Hospital Vall d'Hebron

OTHER

collaborator

Universitat Autonoma de Barcelona

OTHER

lead

Hospital Infantil Universitario Niño Jesús, Madrid, Spain

OTHER