Ignite Creation Date:
2025-12-24 @ 10:46 PM
Ignite Modification Date:
2025-12-31 @ 12:08 AM
Study NCT ID:
NCT04472169
Status:
UNKNOWN
Last Update Posted:
2023-02-10
First Post:
2020-07-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
UPLC-MS/MS Monitoring of Emicizumab Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], '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': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Serum, poor-platelet plasma, whole blood'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-04-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-02', 'completionDateStruct': {'date': '2024-02', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-02-09', 'studyFirstSubmitDate': '2020-07-10', 'studyFirstSubmitQcDate': '2020-07-10', 'lastUpdatePostDateStruct': {'date': '2023-02-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-07-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-02', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Area under the curve ROC of Residual plasma level of emicizumab', 'timeFrame': 'At Week 5 (end of emicizumab loading period)', 'description': 'At least one clinically significant bleeding (defined as any bleeding treated with FVIII, rFVIIa or aPCC) from loading period completion (week 5) to the end of study, an average of 1 year'}], 'secondaryOutcomes': [{'measure': 'Residual plasma level of emicizumab measured by UPLC-MS/MS', 'timeFrame': 'At Week 5 (end of emicizumab loading period)', 'description': 'At least one hemarthrosis from loading period completion (week 5) to the end of study, an average of 1 year'}, {'measure': 'Residual plasma level of emicizumab measured by UPLC-MS/MS', 'timeFrame': 'At each breakthrough bleeding until end of study', 'description': 'Post-traumatic or spontaneous nature of bleeding event'}, {'measure': 'Residual plasma level of emicizumab (UPLC-MS/MS dosing)', 'timeFrame': 'At Week 5 and at each breakthrough bleeding until end of study', 'description': 'Emicizumab FVIII-like activity (chromogenic FVIII BIOPHEN™assay system with emicizumab calibration)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Drug monitoring', 'Severe Hemophilia A', 'Emicizumab'], 'conditions': ['Hemophilia A']}, 'descriptionModule': {'briefSummary': 'Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': '100 patients with severe hemophilia A will be included in the study on 2 french Hemophilia Treatement Centers (CHU Lille and CHU Caen)', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult or child with Clinical diagnosis of severe hemophilia A (FVIII activity \\< 1%) with or without inhibitor\n* Clinical indication to emicizumab therapy\n\nExclusion Criteria:\n\n* Refusal to give informed consent\n* acquired hemophilia A\n* other inherited or acquired bleeding disorder\n* bodyweight \\< 10 kgs'}, 'identificationModule': {'nctId': 'NCT04472169', 'acronym': 'EMICARE', 'briefTitle': 'UPLC-MS/MS Monitoring of Emicizumab Therapy', 'organization': {'class': 'OTHER', 'fullName': 'University Hospital, Lille'}, 'officialTitle': 'Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A', 'orgStudyIdInfo': {'id': '2019_75'}, 'secondaryIdInfos': [{'id': '2020-A00584-35', 'type': 'OTHER', 'domain': 'ID-RCB number,ANSM'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Severe haemophila A patients with or without inhibitors'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Caen', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Yohann Repesse, MD,PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'CHU de Caen', 'geoPoint': {'lat': 49.18585, 'lon': -0.35912}}, {'zip': '59037', 'city': 'Lille', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'role': 'CONTACT', 'phone': '0320445962'}, {'name': 'Antoine Rauch, MD,PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU", 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}], 'centralContacts': [{'name': 'Antoine Rauch, MD,PhD', 'role': 'CONTACT', 'email': 'antoine.rauch@chru-lille.fr', 'phone': '0320445962', 'phoneExt': '+33'}], 'overallOfficials': [{'name': 'Antoine Rauch, MD,PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University Hospital, Lille'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Hospital, Lille', 'class': 'OTHER'}, 'collaborators': [{'name': "Groupement Interrégional de Recherche Clinique et d'Innovation", 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}