Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia
Research type
Research Study
Full title
Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient
IRAS ID
167477
Contact name
Adrian Thrasher
Contact email
Sponsor organisation
Great Ormond street Hospital NHS foundation trust
Eudract number
2014-004272-29
Duration of Study in the UK
1 years, 11 months, 28 days
Research summary
Fanconi anaemia (FA) is a recessive gene disorder disease (with the exception of FA-B), due to a defects in a number of FA genes. FA is principally characterised by bone marrow failure and increased predisposition to cancer. At birth, a patient's blood count is generally normal, but after 5 to 10 years the bone marrow fails, with the average age of onset of haematological disease being 7 years old. About 80% of patients with FA will develop evidence of bone marrow failure (thrombocytopenia, anaemia) in the first decade of life.
Currently, there are no medications specifically indicated for FA and allogeneic haematopoietic transplantation (which involves replacing the patient’s bone marrow with that of a healthy donor) is the only treatment that definitely restores the normal function of the bone marrow in patients with FA. The results from identical familial donor transplant procedure have improved in recent years with some centres reaching over 80% survival at 5 years after the procedure.
Patients are invited to participate in a Phase II clinical trial to evaluate safety and efficacy (effect) of mobilization and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia. If a sufficient number of cells are collected they will be stored in a liquid nitrogen tank and patients will be considered for gene therapy at a later date.
In this study, we plan to use two medicines, one named plerixafor and the other filgrastim in the hope that the haematopoetic stem cells can mobilise from the bone marrow and be harvested (collected) from patients with Fanconi Anaemia. What this means is that before any cell collection takes place, the medicines that you will receive will help promote movement of these stem cells into the blood stream (mobilisation) making collection more accessible.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/0126
Date of REC Opinion
30 Mar 2015
REC opinion
Further Information Favourable Opinion