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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00927134 |
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Date of registration:
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22/06/2009 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
XCGDinChildren |
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Scientific title:
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Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children |
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Date of first enrolment:
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June 2004 |
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Target sample size:
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2 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00927134 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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Switzerland
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Contacts
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Name:
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Reinhard Seger, Prof Dr med |
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Address:
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Telephone:
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Email:
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Affiliation:
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University Children's Hospital Zürich |
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Name:
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Manuel Grez, Dr rer nat |
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Address:
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Telephone:
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Email:
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Affiliation:
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Georg Speyer Research Institute, Frankfurt a.M. |
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Name:
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Janine Reichenbach, PD Dr med |
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Address:
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Telephone:
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Email:
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Affiliation:
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University Children's Hospital Zürich |
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Name:
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Ulrich Siler, Dr rer nat |
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Address:
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Telephone:
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Email:
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Affiliation:
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University Children's Hospital Zürich |
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Key inclusion & exclusion criteria
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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)
Age minimum:
1 Year
Age maximum:
18 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Chronic Granulomatous Disease
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Intervention(s)
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Genetic: retroviral SF71-gp91phox transduced CD34+ cells
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Primary Outcome(s)
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eradication of pre-existing therapy refractory bacterial and/or fungal infections
[Time Frame: 6 months]
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Secondary Outcome(s)
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Reconstitution of ROS production by peripheral blood cells
[Time Frame: 1 month]
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Secondary ID(s)
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PedsZürich_GT05
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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