Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

Plain-language summary

This study used the drugs filgrastim and plerixafor to evaluate the mobilization and collection of CD34+ stem cells in patients with Fanconi anemia, so the cells could later be genetically corrected with a lentiviral vector carrying the FANCA gene. It included patients older than 1 year with Fanconi anemia confirmed by a chromosomal fragility test. It was conducted in 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.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Hospital Universitari Vall d'Hebron Research Institute

Intervention

filgrastim, plerixafor

Start date

9/1/2013

Estimated completion

10/1/2018

Countries

Spain

Related genes

Locations

• Barcelona, Spain
• Madrid, Spain

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Male or female \> 1 year
* diagnosed of Fanconi's anemia confirmed by instability chromosomal test with diepoxy-butane or mitomycin C
* At least one of the following parameters must be higher than these values: Hemoglobin:8,0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3
* Lansky index\> 60%.
* Left ventricular ejection fraction \>50%.
* To grant informed consent in agreement with current law norms
* Women in childbearing age must obtain a negative result in the pregnancy test in serum or urine in the visit of selection and accept the use of suitable contraceptive methods since at least 14 days prior to the first dose of mobilizing treatment until the 14 days following the last

Exclusion Criteria:

* Evidence of myelodysplastic syndromes or leukemia, or cytogenetic abnormalities predicted of these syndromes in bone marrow aspiration. Cytogenetic analyses performed 2 months before starting study are accepted
* Patients with active infection process or any other underlaying severe medical process
* Severe Functional alteration of organs (hepatic, renal, respiratory)(?3), according to National Cancer Institute (NCI CTCAE v3) criteria
* Haematopoietic transplant
* Any disease or concomitant process that is not compatible with the study as per investigator opinion
* Patients not elegible because of an psico-social evaluation
* Patients that received transfusional support during the last 3 months.
* Pregnant or breastfeeding women