Clinical Trial: Gene Therapy for ADA-SCID

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Treatment of ADA-SCID by Gene Therapy on Somatic Cells

Brief Summary: This study investigated the safety and efficacy of different gene therapy approaches for Severe Combined Immunodeficiency (SCID) caused by the deficiency of adenosine deaminase (ADA) enzyme. This is a severe condition that can be cured by HLA-matched sibling donor bone marrow transplantation. Patients were enrolled if no HLA-identical sibling donor was available and the patient showed evidence of failure of enzyme replacement therapy or this treatment was not a long-term available option. The aim of the study was to evaluate the safety and efficacy of the procedure and to identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after retroviral vector mediated ADA gene transfer.

Detailed Summary: This is mono-centric, non-randomized, non-controlled, open label, phase I-II trial that evaluated the safety and efficacy of ADA gene transfer into somatic cells for the treatment of ADA-SCID
Sponsor: IRCCS San Raffaele

Current Primary Outcome: Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Evaluation of extent, kinetic and duration of the engraftment of transduced cells and the potential selective advantage of ADA positive cells
  • Evaluation of efficacy of the administration of autologous PBL/HSC(Clinical, immunological, hematological, microbiological, ADA activity and purine metabolism)
  • To identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after gene therapy


Original Secondary Outcome: Same as current

Information By: IRCCS San Raffaele

Dates:
Date Received: January 8, 2008
Date Started: March 1992
Date Completion:
Last Updated: January 23, 2008
Last Verified: December 2007