Ignite Creation Date:
2025-12-25 @ 2:38 AM
Ignite Modification Date:
2025-12-27 @ 11:30 PM
Study NCT ID:
NCT00927134
Status:
COMPLETED
Last Update Posted:
2011-09-27
First Post:
2009-06-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
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'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2004-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-09', 'completionDateStruct': {'date': '2011-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2011-09-26', 'studyFirstSubmitDate': '2009-06-22', 'studyFirstSubmitQcDate': '2009-06-23', 'lastUpdatePostDateStruct': {'date': '2011-09-27', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2009-06-24', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'eradication of pre-existing therapy refractory bacterial and/or fungal infections', 'timeFrame': '6 months'}], 'secondaryOutcomes': [{'measure': 'Reconstitution of ROS production by peripheral blood cells', 'timeFrame': '1 month'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Chronic Granulomatous Disease in children (=or< 18 years)'], 'conditions': ['Chronic Granulomatous Disease']}, 'referencesModule': {'references': [{'pmid': '16582916', 'type': 'BACKGROUND', '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) children 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': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '1 Year', '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 unrelated donor\n* therapy resistent life threatening infections/organ dysfunction\n* no other treatment options e.g. HSCT\n\nExclusion Criteria:\n\n* \\> 18 years of age\n* HIV infection\n* life expectancy \\> 2 years\n* infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)'}, 'identificationModule': {'nctId': 'NCT00927134', 'acronym': 'XCGDinChildren', 'briefTitle': 'Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children', 'organization': {'class': 'OTHER', 'fullName': 'University of Zurich'}, 'officialTitle': 'Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children', 'orgStudyIdInfo': {'id': 'PedsZürich_GT05'}}, 'armsInterventionsModule': {'interventions': [{'name': 'retroviral SF71-gp91phox transduced CD34+ cells', 'type': 'GENETIC', 'description': 'autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells'}]}, 'contactsLocationsModule': {'locations': [{'zip': 'CH-8032', 'city': 'Zurich', 'country': 'Switzerland', 'facility': "University Children's Hospital", 'geoPoint': {'lat': 47.36667, 'lon': 8.55}}], 'overallOfficials': [{'name': 'Reinhard Seger, Prof Dr med', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "University Children's Hospital, Zurich"}, {'name': 'Janine Reichenbach, PD Dr med', 'role': 'STUDY_CHAIR', 'affiliation': "University Children's Hospital, Zurich"}, {'name': 'Ulrich Siler, Dr rer nat', 'role': 'STUDY_CHAIR', 'affiliation': "University Children's Hospital, Zurich"}, {'name': 'Manuel Grez, Dr rer nat', 'role': 'STUDY_CHAIR', 'affiliation': 'Georg Speyer Research Institute, Frankfurt a.M.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Zurich', 'class': 'OTHER'}, 'collaborators': [{'name': 'Goethe University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}