Ignite Creation Date:
2025-12-24 @ 2:08 PM
Ignite Modification Date:
2026-04-22 @ 7:19 AM
Study NCT ID:
NCT03198195
Status:
UNKNOWN
Last Update Posted:
2017-06-27
First Post:
2017-06-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'releaseDate': '2018-09-25', 'unreleaseDate': '2018-10-08'}, {'releaseDate': '2018-10-08', 'unreleaseDate': '2018-10-12'}, {'resetDate': '2019-02-26', 'releaseDate': '2018-10-12'}], 'estimatedResultsFirstSubmitDate': '2018-09-25'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D014923', 'term': 'Wiskott-Aldrich Syndrome'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008231', 'term': 'Lymphopenia'}, {'id': 'D007970', 'term': 'Leukopenia'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003520', 'term': 'Cyclophosphamide'}], 'ancestors': [{'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}, 'targetDuration': '5 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2015-03-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-06', 'completionDateStruct': {'date': '2020-07-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2017-06-26', 'studyFirstSubmitDate': '2017-06-22', 'studyFirstSubmitQcDate': '2017-06-22', 'lastUpdatePostDateStruct': {'date': '2017-06-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-06-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-03-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Rate of aGvHD', 'timeFrame': '3month', 'description': 'after post transplant cyclophospamide'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Wiskott-Aldrich Syndrome']}, 'descriptionModule': {'briefSummary': 'A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate of GvHD.\n\nThe details of the protocal followed with:\n\n1. Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.\n2. GvHD Prophylaxis:\n\nRabbit antihuman thymocyte globulin 7.5 mg/kg post-transplant cyclophosphamide (CY) (50 mg/kg.d on days +3 and +4) Cyclosporine or tacrolimus, mycophenolate mofetil, on days +5', 'detailedDescription': 'Patients were enrolled in CIP-2015 Protocol at the Capital Institute of Pediatrics (Beijing). The conditioning regimen consisted of fludarabine (40 mg/m2) from days -6 to -3, and Busulfan was administered intravenously for 4 days, from days -5 to -2，using dose targeting based on therapeutic drug monitoring. Thymoglobulin (Sanofi, Cambridge, MA) 7.5 to 10 mg/kg (cumulative dose over 4 days) was administered over 4 days, from days -5 to -2. Bone marrow (BM) and PBSC were infused on day 0, followed by post-transplant CY (50 mg/kg/day, on days +3 and +4). To protect against hemorrhagic cystitis, MESNA (2-mercaptoethane sodium sulfonate) was administered at 150% of the CY dose. Post grafting immunosuppression with mycophenolate mofetil and tacrolimus commenced on day +5 and extended until days +28 and +84, respectively. Tacrolimus was tapered off by day +90 if there was no GVHD.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '10 Years', 'minimumAge': '5 Months', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Patient with Wiskott-Aldrich Syndrome :\n\n1. life-threatening bleeding\n2. strong demand of parents', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n-Patients diagnosed with Wiskott-Aldrich Syndrome with indication of Hematopoietic stem cell transplantation\n\nExclusion Criteria:\n\n* without indication of Hematopoietic stem cell transplantation'}, 'identificationModule': {'nctId': 'NCT03198195', 'briefTitle': 'Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome', 'organization': {'class': 'OTHER_GOV', 'fullName': 'Capital Research Institute of Pediatrics'}, 'officialTitle': 'Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome', 'orgStudyIdInfo': {'id': 'CIP-2015-08'}}, 'armsInterventionsModule': {'interventions': [{'name': 'cyclophosphamide', 'type': 'PROCEDURE', 'description': 'on days +3，+4,using cyclophosphamide 50mg/kg'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Capital Research Institute of Pediatrics', 'class': 'OTHER_GOV'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD,PhD', 'investigatorFullName': 'Yan Yue', 'investigatorAffiliation': 'Capital Research Institute of Pediatrics'}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2018-09-25', 'type': 'RELEASE'}, {'date': '2018-10-08', 'type': 'UNRELEASE'}, {'date': '2018-10-08', 'type': 'RELEASE'}, {'date': '2018-10-12', 'type': 'UNRELEASE'}, {'date': '2018-10-12', 'type': 'RELEASE'}, {'date': '2019-02-26', 'type': 'RESET'}], 'unpostedResponsibleParty': 'Yan Yue, MD,PhD, Capital Research Institute of Pediatrics'}}}}