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Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

NCT02931071 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2020-05-27

No results posted yet for this study

Summary

Fanconi anemia (FA) is a congenital disease characterized by bone marrow failure and increased incidence of malignant tumors. The Project pursue the optimization of the collection of hematopoietic progenitor cells for later use in another clinical trial entitled "Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells mobilized with mozobil and filgrastim, and transduced with a lentiviral vector carrying the FANCA gene (Orphan Drug) for patients with Fanconi Anemia Subtype A ". The objectives of this study are, therefore, to assess the safety and efficacy of CD34+ cells mobilization with mozobil and filgrastim, which is postulated the most efficient for the collection of CD34+ cells from FA patients.

Conditions

  • Fanconi Anemia

Interventions

DRUG

filgrastim

G-CSF (12 μg/Kg/12 h) 8 days.

DRUG

plerixafor

Plerixafor 0,24 mg/kg/day after the fourth day of G-CSF, and until 5 cells CD34+/μL, max 4 doses of plerixafor

Sponsors & Collaborators

  • CIEMAT

    collaborator UNKNOWN

  • CIBERER

    collaborator UNKNOWN

  • Hospital Universitari Vall d'Hebron Research Institute

    lead OTHER

Principal Investigators

  • Cristina Díaz de Heredia, MD, PhD · Hospital Vall d'Hebron

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-09-30
Primary Completion
2018-10-31
Completion
2018-10-31

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 NCT02931071 on ClinicalTrials.gov