Clinical trial NCT02931071: 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
Official title: Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fanconi Anemia
Lead sponsor: Hospital Universitari Vall d'Hebron Research Institute
Sponsor class: OTHER
Collaborators: name: CIEMAT
name: CIBERER
First posted: 2016-10-12
Enrollment: 13
Sex: ALL
Minimum age (years): 2
Maximum age (years): 64
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Fanconi anemia (FA) is a congenital disease characterized by bone marrow failure and increased incidence of malignant tumors. The Project pursue the optimization of the collection of hematopoietic progenitor cells for later use in another clinical trial entitled "Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells mobilized with mozobil and filgrastim, and transduced with a lentiviral vector carrying the FANCA gene (Orphan Drug) for patients with Fanconi Anemia Subtype A ". The objectives of this study are, therefore, to assess the safety and efficacy of CD34+ cells mobilization with mozobil and filgrastim, which is postulated the most efficient for the collection of CD34+ cells from FA patients.


Eligibility criteria: 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

Primary outcomes:
measure: Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)
timeFrame: after 12 months
Secondary outcomes:
measure: Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor
timeFrame: after 8 days
measure: Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years
timeFrame: after 8 days
measure: Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50%
timeFrame: after 8 days
measure: Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years
timeFrame: after 8 days
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Niño Jesus
Study officials:
affiliation: Hospital Vall d'Hebron
name: Cristina Díaz de Heredia, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sevilla J, Navarro S, Rio P, Sanchez-Dominguez R, Zubicaray J, Galvez E, Merino E, Sebastian E, Azqueta C, Casado JA, Segovia JC, Alberquilla O, Bogliolo M, Roman-Rodriguez FJ, Gimenez Y, Larcher L, Salgado R, Pujol RM, Hladun R, Castillo A, Soulier J, Querol S, Fernandez J, Schwartz J, Garcia de Andoin N, Lopez R, Catala A, Surralles J, Diaz-de-Heredia C, Bueren JA. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes. Mol Ther Methods Clin Dev. 2021 Jun 12;22:66-75. doi: 10.1016/j.omtm.2021.06.001. eCollection 2021 Sep 10.