Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)

Disease Information

Descriptive Information
Brief Title † Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
Official Title † A Phase I/II, Non Randomized, Multicenter, Open-label Study of Autologous CD34+ Cells Transduced With the G1XCGD Lentiviral Vector in Patients With X-linked Chronic Granulomatous Disease
Brief Summary X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is caused by an error in a gene that makes part of the immune system. The basic defect lies in specialised white blood cells called phagocytic cells (or phagocytes), which are responsible for protection against infection by destroying invading bacteria and fungi. They do this by pouring large amounts of substances similar to bleach onto these organisms. In CGD, there is a defect in the system that makes the bleach, called the NADPH-oxidase. In X-CGD (which accounts for two thirds of patients), the defect lies in a gene which makes up a critical part of the NADPH-oxidase (known as gp91-phox), and the cells cannot make bleach-like substances. Therefore they kill bacteria and fungi poorly, and the patients suffer from severe and recurrent infections. This also results in inflammation which can damage parts of the body such as the lung and gut. In many cases, patients can be adequately protected from infection by constant intake of antibiotics. However, in others, severe life-threatening infections break through. In some cases, inflammation in the bowel or urinary systems results in blockages which cannot be treated with antibiotics, and which may require the use of other drugs such as steroids. Development of curative treatments for CGD is therefore of great importance.
Detailed Description
Study Phase Phase 1/Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Safety of the procedure as measured by the incidence of adverse events
Secondary Outcome Measure † Normalisation of nutritional status, growth, development, severe infection and/or inflammatory complication which recommended patient's inclusion
Condition † X-Linked Chronic Granulomatous Disease
Intervention † GeneticX vivo gene therapy
Study Arms / Comparison Groups Open label X vivo gene therapy
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 † 5
Start Date † February 2013
Completion Date March 2020
Primary Completion Date March 2020
Eligibility Criteria † Inclusion Criteria: - Male X-CGD patients - Molecular diagnosis confirmed by DNA sequencing - At least one prior ongoing or resistant severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy - No HLA-matched donor available after 3 months search unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable by the investigator Exclusion Criteria: - Contraindication for leukapheresis - Contraindication for administration of conditioning medication - Administration of gammainterferon within 30 days before the infusion of transduced autologous CD34+ cells
Gender Male
Ages 6 Months - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † Germany
Administrative Information
NCT ID † NCT01855685
Organization ID G1XCGD.01
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Genethon
Collaborators ††
Investigators † Principal Investigator: Adrian Thrasher, MD, PHD, Great Ormond Street Hospital NHS Foundation Trust - London - UK
Information Provided By
Verification Date January 2017
First Received Date † May 14, 2013
Last Updated Date February 7, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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