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

• Register: ClinicalTrials.gov
• Last refreshed on: 4 January 2022
• Main ID: NCT02678533
• Date of registration: 05/02/2016
• Prospective Registration: Yes
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor FancoMob
• Scientific title: Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy
• Date of first enrolment: February 10, 2017
• Target sample size: 4
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02678533
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

France

Contacts

• Name: Marina CAVAZZANA, MD, PhD
• Affiliation: AP-HP, Necker hospital

Key inclusion & exclusion 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



Age minimum: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia

Intervention(s)

Drug: Plerixafor

Drug: G-CSF

Primary Outcome(s)

level of CD34+ cells mobilization [Time Frame: from day 5 to day 8 after the first injection of G-CSF]

Secondary Outcome(s)

number of treatment-related adverse events as a measure of tolerability [Time Frame: 30 days after cytapheresis]

Secondary ID(s)

P130103

2014-005264-14

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

EuroFancolen