Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
NCT ID: NCT00927134
Last Updated: 2011-09-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
2 participants
INTERVENTIONAL
2004-06-30
2011-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Interventions
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retroviral SF71-gp91phox transduced CD34+ cells
autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells
Eligibility Criteria
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Inclusion Criteria
* 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
* HIV infection
* life expectancy \> 2 years
* infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)
1 Year
18 Years
MALE
No
Sponsors
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Goethe University
OTHER
University of Zurich
OTHER
Responsible Party
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Principal Investigators
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Reinhard Seger, Prof Dr med
Role: PRINCIPAL_INVESTIGATOR
University Children's Hospital, Zurich
Janine Reichenbach, PD Dr med
Role: STUDY_CHAIR
University Children's Hospital, Zurich
Ulrich Siler, Dr rer nat
Role: STUDY_CHAIR
University Children's Hospital, Zurich
Manuel Grez, Dr rer nat
Role: STUDY_CHAIR
Georg Speyer Research Institute, Frankfurt a.M.
Locations
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University Children's Hospital
Zurich, , Switzerland
Countries
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References
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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.
Other Identifiers
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PedsZürich_GT05
Identifier Type: -
Identifier Source: org_study_id