Clinical Trial: Gene Therapy ADA Deficiency

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

Official Title: Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency

Brief Summary: Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.

Detailed Summary:
Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

Current Primary Outcome: Immunological reconstitution [ Time Frame: 5 years ]

Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Incidence of adverse reactions [ Time Frame: 5 years ]
    Incidence of adverse reactions. 5 year follow up of the last patient enrolled into study
  • Molecular characterisation of gene transfer [ Time Frame: 5 years ]
    Molecular characterisation of gene transfer. 5 year follow up of the last patient enrolled into study
  • Normalisation of nutritional status, growth, and development [ Time Frame: 5 years ]
    Normalisation of nutritional status, growth, and development. 5 year follow up of the last patient enrolled into study


Original Secondary Outcome: Same as current

Information By: Great Ormond Street Hospital for Children NHS Foundation Trust

Dates:
Date Received: January 18, 2011
Date Started: October 2003
Date Completion:
Last Updated: September 11, 2015
Last Verified: September 2015