Gene Therapy for Chronic Granulomatous Disease (CGD)
The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2007 by Johann Wolfgang Goethe University Hospitals.
Recruitment status was Active, not recruiting
Recruitment status was Active, not recruiting
Sponsor:
Johann Wolfgang Goethe University Hospitals
Collaborator:
German Federal Ministry of Education and Research
Information provided by:
Johann Wolfgang Goethe University Hospitals
ClinicalTrials.gov Identifier:
NCT00564759
First received: November 26, 2007
Last updated: November 27, 2007
Last verified: November 2007
| Tracking Information | |||||
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| First Received Date ICMJE | November 26, 2007 | ||||
| Last Updated Date | November 27, 2007 | ||||
| Start Date ICMJE | January 2004 | ||||
| Primary Completion Date | Not Provided | ||||
| Current Primary Outcome Measures ICMJE |
safety, toxicity and feasibility [ Time Frame: 2 years ] | ||||
| Original Primary Outcome Measures ICMJE | Same as current | ||||
| Change History | Complete list of historical versions of study NCT00564759 on ClinicalTrials.gov Archive Site | ||||
| Current Secondary Outcome Measures ICMJE |
Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit [ Time Frame: 2 years ] | ||||
| Original Secondary Outcome Measures ICMJE | Same as current | ||||
| Current Other Outcome Measures ICMJE | Not Provided | ||||
| Original Other Outcome Measures ICMJE | Not Provided | ||||
| Descriptive Information | |||||
| Brief Title ICMJE | Gene Therapy for Chronic Granulomatous Disease | ||||
| Official Title ICMJE | Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease | ||||
| 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)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. |
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| Detailed Description | Not Provided | ||||
| Study Type ICMJE | Interventional | ||||
| Study Phase | Phase 1 Phase 2 |
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| Study Design ICMJE | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label |
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| Condition ICMJE | Granulomatous Disease, Chronic | ||||
| Intervention ICMJE | Drug: retroviral SF71-gp91phox transduced CD34+ cells
autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells |
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| Study Arm (s) | Not Provided | ||||
| Publications * | Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kühlcke 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, Lüthi 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. Epub 2006 Apr 2. | ||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status ICMJE | Active, not recruiting | ||||
| Enrollment ICMJE | 2 | ||||
| Estimated Completion Date | December 2008 | ||||
| Primary Completion Date | Not Provided | ||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Male | ||||
| Ages | 18 Years and older | ||||
| Accepts Healthy Volunteers | No | ||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
| Location Countries ICMJE | Germany | ||||
| Administrative Information | |||||
| NCT Number ICMJE | NCT00564759 | ||||
| Other Study ID Numbers ICMJE | 58/59, DeReG 31, KSG 31 | ||||
| Has Data Monitoring Committee | No | ||||
| Responsible Party | Not Provided | ||||
| Study Sponsor ICMJE | Johann Wolfgang Goethe University Hospitals | ||||
| Collaborators ICMJE | German Federal Ministry of Education and Research | ||||
| Investigators ICMJE |
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| Information Provided By | Johann Wolfgang Goethe University Hospitals | ||||
| Verification Date | November 2007 | ||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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