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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: |
NCT00564759 |
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Date of registration:
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26/11/2007 |
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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 Chronic Granulomatous Disease
CGD |
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Scientific title:
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Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease |
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Date of first enrolment:
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January 2004 |
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Target sample size:
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2 |
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Recruitment status: |
Active, not recruiting |
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URL:
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http://clinicaltrials.gov/show/NCT00564759 |
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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
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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Germany
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Contacts
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Name:
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Dieter Hoelzer, MD, PhD |
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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 Hospital, Frankfurt |
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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 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)
Age minimum:
18 Years
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Granulomatous Disease, Chronic
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Intervention(s)
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Drug: retroviral SF71-gp91phox transduced CD34+ cells
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Primary Outcome(s)
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safety, toxicity and feasibility
[Time Frame: 2 years]
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Secondary Outcome(s)
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Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
[Time Frame: 2 years]
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Secondary ID(s)
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KSG 31
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58/59
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DeReG 31
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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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