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 range
2 Years – 17 Years
Sex
ALL
See this in plain English?
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
level of CD34+ cells mobilization
Timeframe: from day 5 to day 8 after the first injection of G-CSF