Clinical Trial: Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

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

Official Title: Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FA

Brief Summary:

This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .

CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will be inoculated in patients in order to restore their hematopoiesis with genetically corrected stem cells.


Detailed Summary:

The main objective of this open-label Phase I / II clinical trial is to evaluate the safety and therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi's Anemia Subtype A.

The drug to be administered to the patients consists of the cellular product resulting from the transduction of autologous CD34 + cells with the therapeutic lentiviral vector PGK-FANCA.Wpre *.

The dose of cells to infuse in the patients will be that obtained from the transduction process of between 3x10^5 and 4x10^6 CD34 + cells / kg of patient body weight.

The cells will be infused intravenously in a single dose, after complete the transduction process.

Follow-up period: 2 years after infusion of transduced cells. However, patients will be monitored outside the clinical trial over a 10-year period.

Follow-up of the grafted transduced cells will be performed on peripheral blood and bone marrow samples.


Sponsor: Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Current Primary Outcome:

  • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Up to 2 years after infusion of transduced cells ]
    All adverse events will be registered for 2 years from infusion of transduced cells
  • Proportion of patients with at least 0.1 copy of the therapeutic vector per nucleated bone marrow or peripheral blood cells two years after infusion. [ Time Frame: 2 years after infusion of transduced cells ]
    Detection of at least 0.1 copy of the therapeutic vector per nucleated bone marrow cell or peripheral blood cells two years after infusion.


Original Primary Outcome: Same as current

Current Secondary Outcome: Proportion of patients with clinical hematological response after the infusion of autologous CD34 + cells transduced with the therapeutic lentiviral vector [ Time Frame: 2 years after infusion of transduced cells ]

Proportion of patients with clinical hematological response (improvement of cell blood counts at least in one hematological lineage).


Original Secondary Outcome: Same as current

Information By: Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Dates:
Date Received: April 26, 2017
Date Started: January 2016
Date Completion: February 2019
Last Updated: May 16, 2017
Last Verified: May 2017