Clinical Trial: 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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 Transdu

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.

Detailed Summary:
Sponsor: Hospital Universitari Vall d'Hebron Research Institute

Current Primary Outcome: Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0) [ Time Frame: after 12 months ]

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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor [ Time Frame: after 8 days ]
    mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter
  • Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years [ Time Frame: after 8 days ]
    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
  • Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50% [ Time Frame: after 8 days ]
    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%
  • Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years [ Time Frame: after 8 days ]
    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


Original Secondary Outcome: Same as current

Information By: Hospital Universitari Vall d'Hebron Research Institute

Dates:
Date Received: September 6, 2016
Date Started: September 2013
Date Completion: September 2018
Last Updated: October 10, 2016
Last Verified: October 2016