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 ]
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