Yükleniyor... / Loading...
This study examines collecting stem cells from the blood using G-CSF and Plerixafor in children with Fanconi anemia. Children aged 2 to 17 who are being considered for transplant and have no suitable HLA-matched sibling donor could take part. It was conducted in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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