Clinical Trial: Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)
Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional
Official Title: Phase I/II Gene Therapy Protocol for X-Linked Chronic Granulomatous Disease
Brief Summary:
Chronic Granulomatous Disease (CGD) is a rare inherited disorder in which patients suffer from severe infection and inflammation. The first indication of disease usually appears in early childhood. The basic defect found to be lie in specialised white blood cells called phagocytic cells, which are responsible for engulfing and destroying germs. In CGD, there is a defect in an enzyme (known as NADPH-oxidase) that is responsible for generating bleach like substances that are important for killing some important germs. In the form of the disease known as X-CGD (which accounts for two thirds of patients), there are defined mistakes in a gene called gp91-phox, which is a key part of the NADPH-oxidase.
In many cases, patients can be protected from infection by constant intake of antibiotics. However, in others potential life-threatening infections break through. In some cases patients also develop serious inflammation requiring high doses of drugs such as steroids. CGD can be cured by bone marrow transplant, but the best results are available when there is matched donor available. Transplant from unmatched donor have a much worse outcome.
Gene therapy of CGD can be performed by introducing a normal copy of human gp91-phox gene into the blood forming stem cells of patients' bone marrow by using a gene carrier (in this study called lentiviral vector). After treatment of the bone marrow cells in a specialised laboratory are given back to the patient and will grow into functional phagocytic cells.
Detailed Summary:
Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
Current Primary Outcome: Overall survival following gene therapy [ Time Frame: 3 years follow up ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Reduction in frequency of infections [ Time Frame: evaluated from 1st year after treatment by clinical history, complete physical examination, haematological and microbiological tests ]
- Long term immune reconstitution [ Time Frame: 3 years follow up ]
Original Secondary Outcome: Same as current
Information By: Great Ormond Street Hospital for Children NHS Foundation Trust
Dates:
Date Received: June 23, 2011
Date Started: November 2011
Date Completion: November 2016
Last Updated: June 1, 2012
Last Verified: June 2012
