Trial | NCT00599781  Report issue

Title

Gene Therapy for ADA-SCID
Treatment of ADA-SCID by Gene Therapy on Somatic Cells

  • Phase

    Phase 1/Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Study Participants

    8
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.
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
Study Started
Mar 31
1992
Primary Completion
Jul 31
2006
Study Completion
Jan 31
2007
Last Update
Jan 24
2008
Estimate

Genetic gene transduced PBL and/or gene transduced HSC

infusions of autologous PBL and/or HSC transduced with retroviral vectors encoding ADA

  • Other names: gene therapy

PBL/HSC Experimental

Criteria 

Inclusion Criteria:

Lack of HLA-identical sibling donor and
Evidence of failure of the enzyme replacement treatment after >6 months or
PEG-ADA is not available as a life long option

Exclusion Criteria:

HLA identical bone marrow sibling donor
HIV infection
Malignancy
No Results Posted