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

Phase 1

Completed

• Conditions: Chronic Granulomatous Disease

• Registration Number: NCT00927134
• Lead Sponsor: University of Zurich

Brief Summary

The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-06
• Study First Submitted: 2009-06-22
• Study First Submitted QC: 2009-06-23
• Study First Posted: 2009-06-24
• Primary Completion: 2010-12
• Status Verified: 2011-09
• Study Completion: 2011-09
• Last Update Submitted: 2011-09-26
• Last Update Posted: 2011-09-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 2

Inclusion Criteria

• x-linked Chronic Granulomatous Disease
• history of life-threatening severe infections
• no HLA-matched related or unrelated donor
• therapy resistent life threatening infections/organ dysfunction
• no other treatment options e.g. HSCT

Exclusion Criteria

• > 18 years of age
• HIV infection
• life expectancy > 2 years
• infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
eradication of pre-existing therapy refractory bacterial and/or fungal infections	6 months

Secondary Outcome Measures

Name	Time	Method
Reconstitution of ROS production by peripheral blood cells	1 month

Trial Locations

Locations (1)

University Children's Hospital; 🇨🇭 Zürich, Switzerland