NCT01279720

Gene Therapy ADA Deficiency

Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Adenosine Deaminase Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Intravenous infusion of transduced cells	Intravenous infusion of transduced cells

Timeline

• Start date: 2003-10-01
• Primary completion: 2013-11-01
• Completion: 2013-11-01
• First posted: 2011-01-19
• Last updated: 2015-09-14

Locations

1 site across 1 country: United Kingdom