Ignite Creation Date:
2025-12-24 @ 2:46 PM
Ignite Modification Date:
2026-03-01 @ 1:44 PM
Study NCT ID:
NCT00564759
Status:
UNKNOWN
Last Update Posted:
2007-11-28
First Post:
2007-11-26
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy for Chronic Granulomatous Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006105', 'term': 'Granulomatous Disease, Chronic'}], 'ancestors': [{'id': 'D010585', 'term': 'Phagocyte Bactericidal Dysfunction'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2004-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2007-11', 'completionDateStruct': {'date': '2008-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2007-11-27', 'studyFirstSubmitDate': '2007-11-26', 'studyFirstSubmitQcDate': '2007-11-27', 'lastUpdatePostDateStruct': {'date': '2007-11-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2007-11-28', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'safety, toxicity and feasibility', 'timeFrame': '2 years'}], 'secondaryOutcomes': [{'measure': 'Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit', 'timeFrame': '2 years'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['CGD'], 'conditions': ['Granulomatous Disease, Chronic']}, 'referencesModule': {'references': [{'pmid': '16582916', 'type': 'RESULT', '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.'}]}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* x-linked Chronic Granulomatous Disease\n* history of life-threatening severe infections\n* no HLA-matched related or non-related donor\n* therapy resistent life threatening infections/organ dysfunction\n* no other treatment options e.g. BMT\n\nExclusion Criteria:\n\n* \\< 18 years of age\n* HIV infection\n* life expectancy \\> 2 years\n* infections treatable by conventional therapy (antibiotics, allogeneic granulocytes)'}, 'identificationModule': {'nctId': 'NCT00564759', 'acronym': 'CGD', 'briefTitle': 'Gene Therapy for Chronic Granulomatous Disease', 'organization': {'class': 'OTHER', 'fullName': 'Johann Wolfgang Goethe University Hospital'}, 'officialTitle': 'Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease', 'orgStudyIdInfo': {'id': '58/59'}, 'secondaryIdInfos': [{'id': 'DeReG 31'}, {'id': 'KSG 31'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'retroviral SF71-gp91phox transduced CD34+ cells', 'type': 'DRUG', 'description': 'autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells'}]}, 'contactsLocationsModule': {'locations': [{'zip': '60596', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'University Hospital, Hematology', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}], 'overallOfficials': [{'name': 'Dieter Hoelzer, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Goethe University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Johann Wolfgang Goethe University Hospital', 'class': 'OTHER'}, 'collaborators': [{'name': 'German Federal Ministry of Education and Research', 'class': 'OTHER_GOV'}]}}}