Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children (XCGDinChildren)
Primary Purpose
Chronic Granulomatous Disease
Status
Completed
Phase
Phase 1
Locations
Switzerland
Study Type
Interventional
Intervention
retroviral SF71-gp91phox transduced CD34+ cells
Sponsored by
About this trial
This is an interventional treatment trial for Chronic Granulomatous Disease focused on measuring Chronic Granulomatous Disease in children (=or< 18 years)
Eligibility Criteria
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)
Sites / Locations
- University Children's Hospital
Outcomes
Primary Outcome Measures
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Secondary Outcome Measures
Reconstitution of ROS production by peripheral blood cells
Full Information
NCT ID
NCT00927134
First Posted
June 22, 2009
Last Updated
September 26, 2011
Sponsor
University of Zurich
Collaborators
Goethe University
1. Study Identification
Unique Protocol Identification Number
NCT00927134
Brief Title
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
Acronym
XCGDinChildren
Official Title
Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children
Study Type
Interventional
2. Study Status
Record Verification Date
September 2011
Overall Recruitment Status
Completed
Study Start Date
June 2004 (undefined)
Primary Completion Date
December 2010 (Actual)
Study Completion Date
September 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Zurich
Collaborators
Goethe University
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Granulomatous Disease
Keywords
Chronic Granulomatous Disease in children (=or< 18 years)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Genetic
Intervention Name(s)
retroviral SF71-gp91phox transduced CD34+ cells
Intervention Description
autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells
Primary Outcome Measure Information:
Title
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Reconstitution of ROS production by peripheral blood cells
Time Frame
1 month
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Reinhard Seger, Prof Dr med
Organizational Affiliation
University Children's Hospital, Zurich
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Janine Reichenbach, PD Dr med
Organizational Affiliation
University Children's Hospital, Zurich
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Ulrich Siler, Dr rer nat
Organizational Affiliation
University Children's Hospital, Zurich
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Manuel Grez, Dr rer nat
Organizational Affiliation
Georg Speyer Research Institute, Frankfurt a.M.
Official's Role
Study Chair
Facility Information:
Facility Name
University Children's Hospital
City
Zürich
ZIP/Postal Code
CH-8032
Country
Switzerland
12. IPD Sharing Statement
Citations:
PubMed Identifier
16582916
Citation
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006 Apr;12(4):401-9. doi: 10.1038/nm1393. Epub 2006 Apr 2.
Results Reference
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Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
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