Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)

Disease Information

Descriptive Information
Brief Title † Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)
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 Description
Study Phase Phase 1/Phase 2
Study Type † Interventional
Study Design † Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure † Overall survival following gene therapy
Secondary Outcome Measure † Reduction in frequency of infections
Condition † Granulomatous Disease, Chronic, X-linked, Variant
Intervention † GeneticpCCLchimGp91s lentiviral vector transduced CD34+ cells infusion
Study Arms / Comparison Groups pCCLchimGp91s lentiviral vector transduced CD34+ cells pCCLchimGp91s lentiviral vector transduced CD34+ cells will be infused in a volume of 50-100 mls intravenously over 30-45 minutes
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Genetic
Estimated Enrollment † 0
Start Date † November 2011
Completion Date November 2016
Primary Completion Date November 2016
Eligibility Criteria † Inclusion Criteria: - History of at least one severe infection requiring hospitalisation despite standard antimicrobial prophylaxis and/or inflammation complications including one of the following: Oesophageal obstruction, gastric outlet obstruction, bladder outlet obstruction or colitis - Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase - Parental/Guardian and where appropriate Child's signed consent/assent Exclusion Criteria: - 10/10 HLA identical (A,B,C,DR,DQ) family or unrelated or cord blood donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure - Contraindication for leukapheresis (anaemia Hb
Gender Male
Ages N/A - 30 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United Kingdom
Administrative Information
NCT ID † NCT01381003
Organization ID 11-MI-03
Secondary IDs ††
Responsible Party
Study Sponsor † Great Ormond Street Hospital for Children NHS Foundation Trust
Collaborators ††
Investigators † Principal Investigator: Adrian Thrasher, Professor, Great Ormond Street Hospital for Children NHS Foundation Trust
Information Provided By
Verification Date June 2012
First Received Date † June 23, 2011
Last Updated Date June 1, 2012
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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