- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00564759
Gene Therapy for Chronic Granulomatous Disease (CGD)
November 27, 2007 updated by: Johann Wolfgang Goethe University Hospital
Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease
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)patients 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
Unknown
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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Frankfurt, Germany, 60596
- University Hospital, Hematology
-
-
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
18 years and older (Adult, Older Adult)
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 non-related donor
- therapy resistent life threatening infections/organ dysfunction
- no other treatment options e.g. BMT
Exclusion Criteria:
- < 18 years of age
- HIV infection
- life expectancy > 2 years
- infections treatable by conventional therapy (antibiotics, 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
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
safety, toxicity and feasibility
Time Frame: 2 years
|
2 years
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
Time Frame: 2 years
|
2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Collaborators
Investigators
- Principal Investigator: Dieter Hoelzer, MD, PhD, University Hospital Frankfurt
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
January 1, 2004
Study Completion (Anticipated)
December 1, 2008
Study Registration Dates
First Submitted
November 26, 2007
First Submitted That Met QC Criteria
November 27, 2007
First Posted (Estimate)
November 28, 2007
Study Record Updates
Last Update Posted (Estimate)
November 28, 2007
Last Update Submitted That Met QC Criteria
November 27, 2007
Last Verified
November 1, 2007
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 58/59
- DeReG 31
- KSG 31
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.
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