Ignite Creation Date:
2025-12-24 @ 9:07 PM
Ignite Modification Date:
2025-12-30 @ 10:40 PM
Study NCT ID:
NCT01340404
Status:
COMPLETED
Last Update Posted:
2016-12-05
First Post:
2011-04-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D020521', 'term': 'Stroke'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D033581', 'term': 'Stem Cell Transplantation'}], 'ancestors': [{'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D014180', 'term': 'Transplantation'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 63}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-11', 'completionDateStruct': {'date': '2015-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-12-02', 'studyFirstSubmitDate': '2011-04-21', 'studyFirstSubmitQcDate': '2011-04-21', 'lastUpdatePostDateStruct': {'date': '2016-12-05', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-04-22', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'cognitive dysfunction', 'timeFrame': '12 months', 'description': "the scale of cognitive dyfunction depends on the child's age : definition of dysfunction uses the WISC-4, WPPSI-4 or WAIS-3 according to age"}], 'primaryOutcomes': [{'measure': 'Cerebral vasculopathy', 'timeFrame': '1 year', 'description': 'velocity in the artery with highest velocity'}], 'secondaryOutcomes': [{'measure': 'ischemic lesions on magnetic resonance imaging (MRI)', 'timeFrame': '1 year', 'description': 'percentage of patients with ischemic lesions on magnetic resonance imaging (MRI)'}, {'measure': 'stenoses on magnetic resonance imaging (MRI)', 'timeFrame': '1 year', 'description': 'percentage of patients with stenoses on magnetic resonance imaging (MRI)'}, {'measure': 'normalisation of arterial velocities', 'timeFrame': '12 months', 'description': 'percentage of patients at 1 year with velocities normalization on transcranial doppler (TCD)(\\< 170 cm/sec)'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Sickle Cell Anemia', 'Cerebrovascular Accident']}, 'referencesModule': {'references': [{'pmid': '30667500', 'type': 'DERIVED', 'citation': 'Bernaudin F, Verlhac S, Peffault de Latour R, Dalle JH, Brousse V, Petras E, Thuret I, Paillard C, Neven B, Galambrun C, Divialle-Doumdo L, Pondarre C, Guitton C, Missud F, Runel C, Jubert C, Elana G, Ducros-Miralles E, Drain E, Taieb O, Arnaud C, Kamdem A, Malric A, Elmaleh-Berges M, Vasile M, Leveille E, Socie G, Chevret S; DREPAGREFFE Trial Investigators. Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia. JAMA. 2019 Jan 22;321(3):266-276. doi: 10.1001/jama.2018.20059.'}, {'pmid': '28821470', 'type': 'DERIVED', 'citation': 'Chevret S, Verlhac S, Ducros-Miralles E, Dalle JH, de Latour RP, de Montalembert M, Benkerrou M, Pondarre C, Thuret I, Guitton C, Lesprit E, Etienne-Julan M, Elana G, Vannier JP, Lutz P, Neven B, Galambrun C, Paillard C, Runel C, Jubert C, Arnaud C, Kamdem A, Brousse V, Missud F, Petras M, Doumdo-Divialle L, Berger C, Freard F, Taieb O, Drain E, Elmaleh M, Vasile M, Khelif Y, Bernaudin M, Chadebech P, Pirenne F, Socie G, Bernaudin F. Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404). Contemp Clin Trials. 2017 Nov;62:91-104. doi: 10.1016/j.cct.2017.08.008. Epub 2017 Aug 15.'}]}, 'descriptionModule': {'briefSummary': 'The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients.', 'detailedDescription': 'The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD)are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients. Sixty-three patients will be enrolled, namely 21 sickle-cell patients in the "SCT" group and 42 in the "Transfusion Program (TP)" group'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '15 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Sickle cell anemia patients (SS/Sb0)\n* \\< 15 years old\n* History of abnormal transcranial doppler (TCD) (≥ 200 cm/sec)\n* Siblings from the same parental couple\n* Parents amenable to Human Leucocyte Antigen (HLA) typing, SCT if an HLA-identical sibling is available or to long-term transfusion program\n\nExclusion Criteria:\n\n* Sickle cell patients older than 15 years\n* no history of abnormal transcranial doppler (TCD) (≥ 200 cm/sec)\n* no sibling from the same parental couple\n* parents averse to HLA typing, SCT if an HLA-identical sibling is available or to long-term transfusion program'}, 'identificationModule': {'nctId': 'NCT01340404', 'acronym': 'DREPAGREFFE', 'briefTitle': 'Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy', 'organization': {'class': 'OTHER', 'fullName': 'Assistance Publique - Hôpitaux de Paris'}, 'officialTitle': 'A Multicenter Study Comparing the Results of Allogeneic Stem Cell Genoidentical in Children With Sickle Cell Anemia and Cerebral Vascular Disease Detected by Transcranial Doppler', 'orgStudyIdInfo': {'id': 'P071247'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Stem Cell Transplantation', 'interventionNames': ['Procedure: Stem cell transplantation']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Transfusion program', 'interventionNames': ['Procedure: Transfusion program']}], 'interventions': [{'name': 'Stem cell transplantation', 'type': 'PROCEDURE', 'description': 'Stem cell transplantation', 'armGroupLabels': ['Stem Cell Transplantation']}, {'name': 'Transfusion program', 'type': 'PROCEDURE', 'description': 'Transfusion program', 'armGroupLabels': ['Transfusion program']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Créteil', 'country': 'France', 'facility': 'CHIC', 'geoPoint': {'lat': 48.79266, 'lon': 2.46569}}], 'overallOfficials': [{'name': 'Françoise Bernaudin, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Assistance Publique - Hôpitaux de Paris'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Assistance Publique - Hôpitaux de Paris', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}