Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children (XCGDinChildren)
September 26, 2011 updated by: University of Zurich
Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
2
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Zürich, Switzerland, CH-8032
- University Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 18 years (ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: SINGLE_GROUP
- Masking: NONE
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Time Frame: 6 months
|
6 months
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
Reconstitution of ROS production by peripheral blood cells
Time Frame: 1 month
|
1 month
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Reinhard Seger, Prof Dr med, University Children's Hospital, Zurich
- Study Chair: Janine Reichenbach, PD Dr med, University Children's Hospital, Zurich
- Study Chair: Ulrich Siler, Dr rer nat, University Children's Hospital, Zurich
- Study Chair: Manuel Grez, Dr rer nat, Georg Speyer Research Institute, Frankfurt a.M.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
June 1, 2004
Primary Completion (ACTUAL)
December 1, 2010
Study Completion (ACTUAL)
September 1, 2011
Study Registration Dates
First Submitted
June 22, 2009
First Submitted That Met QC Criteria
June 23, 2009
First Posted (ESTIMATE)
June 24, 2009
Study Record Updates
Last Update Posted (ESTIMATE)
September 27, 2011
Last Update Submitted That Met QC Criteria
September 26, 2011
Last Verified
September 1, 2011
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- PedsZürich_GT05
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.