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Gene Therapy for ADA-SCID

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ClinicalTrials.gov Identifier: NCT00599781
Recruitment Status : Completed
First Posted : January 24, 2008
Last Update Posted : January 24, 2008
Sponsor:
Collaborator:
Information provided by:

January 8, 2008
January 24, 2008
January 24, 2008
March 1992
July 2006   (Final data collection date for primary outcome measure)
Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene
Same as current
No Changes Posted
  • 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
Same as current
Not Provided
Not Provided
 
Gene Therapy for ADA-SCID
Treatment of ADA-SCID by Gene Therapy on Somatic Cells
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
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Severe Combined Immunodeficiency Syndrome
Genetic: gene transduced PBL and/or gene transduced HSC
infusions of autologous PBL and/or HSC transduced with retroviral vectors encoding ADA
Other Name: gene therapy
Experimental: PBL/HSC
Intervention: Genetic: gene transduced PBL and/or gene transduced HSC

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
8
January 2007
July 2006   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
 
 
NCT00599781
150291
No
Not Provided
Not Provided
Claudio Bordignon, IRCCS San Raffaele
IRCCS San Raffaele
Fondazione Telethon
Principal Investigator: Bordignon Claudio, MD IRCCS San Raffaele
IRCCS San Raffaele
December 2007

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP