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

Fanconi Anemia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02931071
• Other study ID #: FANCOSTEM-1
• Secondary ID: 2011-006197-88
• Status: Completed
• Phase: Phase 2
• Start date: September 2013
• Est. completion date: October 2018

Study information

• Verified date: May 2020
• Source: Hospital Universitari Vall d'Hebron Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: October 2018
• Est. primary completion date: October 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 64 Years

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

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Syndrome

Intervention

Drug:
• filgrastim
G-CSF (12 µg/Kg/12 h) 8 days.
• plerixafor
Plerixafor 0,24 mg/kg/day after the fourth day of G-CSF, and until 5 cells CD34+/µL, max 4 doses of plerixafor

Locations

Country	Name	City	State
Spain	Hospital Universitari Vall d'Hebron	Barcelona
Spain	Hospital Infantil Universitario Niño Jesus	Madrid

Sponsors (3)

Lead Sponsor	Collaborator
Hospital Universitari Vall d'Hebron Research Institute	CIBERER, CIEMAT

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)	At a year (± 30 days) after the last apheresis, a complete physical examination, blood cell count, basic biochemistry and bone marrow aspirate will be done to the patient in order to control their general health status.	after 12 months
Secondary	Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor	mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter	after 8 days
Secondary	Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years	the CD34+ cell collection protocol will be determined by the percentage of patients who reach at least one million CD34 + cells per kilogram of body weight projected to 5 years after the mobilization process	after 8 days
Secondary	Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50%	the of CD34+ cell selection process will be determined by the proportion of immunoselected samples where the recovery of CD34 + cells is at least 50%, and where the final percentage of CD34+ cells is at least 50%	after 8 days
Secondary	Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years	will be determined by the percentage of patients who reach at least one million CD34 + cells per kilo of weight projected to 5 years after the immunomagnetic selection process of all the collected cells	after 8 days