Clinical Trial: Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent T

Brief Summary: The purpose of this study is to assess the feasibility of Plerixafor used in combination with G-CSF (Granulocyte Colony Stimulating Factor) in 5 Fanconi anemia patients to mobilize and collect a sufficient number of peripheral blood CD34+ cells for peripheral blood apheresis, for further gene therapy study.

Detailed Summary:

Fanconi anemia is an autosomal recessive disease with an average survival of around 24 years old. The number of cells producted by bone marrow decreases around 5-10 years old. Hematological symptoms occur around 7 years old. 80% of patients with Fanconi anemia have clinical signs of bone marrow failure in the first decade of life. Generally macrocytosis is the first noticeable sign. Then it leads to thrombocytopenia, anemia and pancytopenia.

Epidemiologic studies show that nearly all of the patients will have medullar aplasia before 40 years old, which is then the first cause of mortality.

It must be emphasized that these complications may occur simultaneously for the same patient, so joint therapeutic intervention is needed.

There is no basic treatment. Some currently used treatments cure cytopenias. These treatments involve blood transfusion, oral androgen, hematopoietic growth factor administration, such as Epo and G-CSF to treat anemia and neutropenia. These treatments are not curative. Hematopoietic stem cell transplantation is the only treatment able to restore permanently hematopoiesis. However, this treatment leads to a high level risk of developing solid tumors and other complications.

All these data justify of developing a stem cells gene therapy treatment using a lentiviral vector expressing wild-type FANCA gene under CIBER promoter.

Three studies have shown the potential number of cells to be mobilized in patients with Fanconi anemia.

The aim is first, to show if administering G-CSF with plerixafor may lead to collect enough cells to potentially perform a gene therapy graft. Secondly the study will assess the toler
Sponsor: Assistance Publique - Hôpitaux de Paris

Current Primary Outcome: level of CD34+ cells mobilization [ Time Frame: from day 5 to day 8 after the first injection of G-CSF ]

Original Primary Outcome: level of CD34+ cells mobilization [ Time Frame: 8 days ]

kinetic from Day 5 to Day 8 after the first injection of G-CSF


Current Secondary Outcome: number of treatment-related adverse events as a measure of tolerability [ Time Frame: 30 days after cytapheresis ]

Occurrence of adverse effect due to G-CSF and plerixafor administration


Original Secondary Outcome: Same as current

Information By: Assistance Publique - Hôpitaux de Paris

Dates:
Date Received: February 5, 2016
Date Started: April 2016
Date Completion: May 2019
Last Updated: April 11, 2016
Last Verified: April 2016