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

• Status: Completed
• Trial end date: 2019-05-03
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborator: EuroFancolen
• Treatments: JM 3100; Lenograstim; Plerixafor

Criteria

Inclusion Criteria:

• Patient with Fanconi anemia

• Patient from 2 to 17 years old

• Potential indication for allogenic bone arrow graft without HLA-identical brotherhood available

• Patient's weight >10kg

• Treated and followed for at least the previous two years in a specialized center where they got a full assessment of their disease

• For women of childbearing age, not pregnant and use of an effective contraception during the entire participation in the research.

• Affiliated or beneficiary of an health insurance regimen

• Informed and signed consent

Exclusion Criteria:

• Patient unable to follow the visits required by the protocol

• Positive serology for HIV-1/2, HTLV-1/2, HCV and HbS

• Bacterial, viral, fungal or parasitic active infection with clinical signs

• Personal history of cancer, myeloproliferative hematopathy or immune deficiency

• Heart failure and / or heart rhythm disorder

• History of allogeneic graft of hematopoietic stem cells

• Patient with an HLA-identical brotherhood donor available

• Myelodysplasia diagnose on myelogram

• Cytogenetic abnormality on karyotype

• Malignant solid tumor

• Documented spontaneous genetic reversion of medullary process

• Diagnosis of a psychiatric disorder that could compromise his/her ability to participate in the study

• Any disorder according to the investigator, that could compromise the ability of patient to give his writing consent and/or to comply with requiring study's procedures

• Current Pregnancy

• Heart, kidney or liver failure

• Current participation in another interventional clinical trial

• Patient under Medical Assistance State

• Hypersensitivity to plerixafor or any excipient contained in MOZOBIL®

• Hypersensitivity to filgrastim or any of its' excipient