Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using … (NCT02678533) | Clinical Trial Compass
CompletedPhase 1/2
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor
France4 participantsStarted 2017-02-10
Plain-language 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.
Who can participate
Age range2 Years – 17 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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, kidn…
What they're measuring
1
level of CD34+ cells mobilization
Timeframe: from day 5 to day 8 after the first injection of G-CSF