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

NCT01381003 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2012-06-04

No results posted yet for this study

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.

Conditions

  • Granulomatous Disease, Chronic, X-linked, Variant

Interventions

GENETIC

pCCLchimGp91s lentiviral vector transduced CD34+ cells infusion

pCCLchimGp91s lentiviral vector transduced CD34+ cells will be infused in a volume of 50-100 mls intravenously over 30-45 minutes

Sponsors & Collaborators

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    lead OTHER

Principal Investigators

  • Adrian Thrasher, Professor · Great Ormond Street Hospital for Children NHS Foundation Trust

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
30 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-11-30
Primary Completion
2016-11-30
Completion
2016-11-30

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01381003 on ClinicalTrials.gov