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Gene Therapy for ADA-SCID

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00599781
Recruitment Status : Completed
First Posted : January 24, 2008
Last Update Posted : January 24, 2008
Sponsor:
Collaborator:
Fondazione Telethon
Information provided by:
IRCCS San Raffaele

Tracking Information
First Submitted Date  ICMJE January 8, 2008
First Posted Date  ICMJE January 24, 2008
Last Update Posted Date January 24, 2008
Study Start Date  ICMJE March 1992
Actual Primary Completion Date July 2006   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 23, 2008)
Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2008)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Gene Therapy for ADA-SCID
Official Title  ICMJE 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 Description 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Severe Combined Immunodeficiency Syndrome
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: PBL/HSC
Intervention: Genetic: gene transduced PBL and/or gene transduced HSC
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 23, 2008)
8
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE January 2007
Actual Primary Completion Date July 2006   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00599781
Other Study ID Numbers  ICMJE 150291
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Claudio Bordignon, IRCCS San Raffaele
Study Sponsor  ICMJE IRCCS San Raffaele
Collaborators  ICMJE Fondazione Telethon
Investigators  ICMJE
Principal Investigator: Bordignon Claudio, MD IRCCS San Raffaele
PRS Account IRCCS San Raffaele
Verification Date December 2007

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