Ignite Creation Date:
2025-12-25 @ 1:08 AM
Ignite Modification Date:
2025-12-27 @ 1:42 AM
Study NCT ID:
NCT05263193
Status:
COMPLETED
Last Update Posted:
2022-12-14
First Post:
2022-02-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C585343', 'term': 'caplacizumab'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'CASE_CONTROL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-06-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-12', 'completionDateStruct': {'date': '2022-10-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-12-13', 'studyFirstSubmitDate': '2022-02-22', 'studyFirstSubmitQcDate': '2022-02-22', 'lastUpdatePostDateStruct': {'date': '2022-12-14', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-03-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-10-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Platelet count response', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'defined as time from caplacizumab initiation to initial platelet count ≥ 150×109/L with subsequent stop of daily plasma exchange (PE) within 5 days'}, {'measure': 'Proportion of subjects with refractory iTTP', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'defined as lack of doubling of platelet count after four days of caplacizumab treatment and a lactate dehydrogenase (LDH) level that remained above the upper limit of normal (ULN) range'}, {'measure': 'Proportion of subjects with recurrent disease', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'Proportion of subjects with iTTP exacerbation (defined as recurrence within 30 days after last PE) and Proportion of subjects with iTTP relapse (defined as recurrence more than 30 days after last PE)'}, {'measure': 'Time to normalization of organ damage marker levels', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'Defined asLDH ≤ 2 x ULN, Serum creatinine ≤ 1 x ULN, Cardiac troponin I ≤ 1 x ULN'}, {'measure': 'Total duration of hospitalization stays', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab'}, {'measure': 'Duration of intensive care unit (ICU) stay', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab'}, {'measure': 'Duration of therapeutic PE', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab'}, {'measure': 'Proportion of patients achieving clinical response', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'defined as a normal platelet countand LDH \\< 2 ULN for at least 48 hours following initial normalization or response of platelet count'}, {'measure': 'Time to ADAMTS13 activity ≥ 20%', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'where available and feasible'}, {'measure': 'Number of participants with Adverse event', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'including serious adverse events'}], 'secondaryOutcomes': [{'measure': 'Treatment pattern of caplacizumab therapy', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab', 'description': 'Dosing and duration'}, {'measure': 'Types and duration of concomitant medications', 'timeFrame': 'From index date up to 12 weeks after last dose of caplacizumab'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Immune-mediated Thrombocytopenic Purpura']}, 'descriptionModule': {'briefSummary': 'The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP.', 'detailedDescription': "Pediatric patients who received caplacizumab will be identified for enrollment in the chart review. The eligibility period starts on August 30, 2018 in the United Kingdom (UK) and France and February 6, 2019 for the United States (US).\n\nData collection is fully retrospective and will be anchored to the patient's index event date. The index event date is defined as the date the patient initiated caplacizumab treatment. The study period begins at the index date and ends at the earliest date of chart abstraction initiation, 12 weeks after last dose of caplacizumab treatment, date of death, or loss to follow-upwhich ever comes first ."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Pediatric patients with Immune-mediated Thrombotic Thrombocytopenic Purpura treated with Caplacizumab', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patient's aged ≤18 years at start of caplacizumab treatment initiation\n* Patient has a diagnosis of iTTP documented in the medical records\n* Patient was treated with caplacizumab within the eligibility period\n\nExclusion Criteria:\n\n* Patient declined use of data for study (where local regulations require patient notification of planned study)\n* Patient's medical chart is missing or not retrievable"}, 'identificationModule': {'nctId': 'NCT05263193', 'briefTitle': 'Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab', 'orgStudyIdInfo': {'id': 'OBS17325'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Caplacizumab', 'type': 'DRUG', 'otherNames': ['Cablivi®'], 'description': 'as in real world practice'}]}, 'contactsLocationsModule': {'locations': [{'zip': '91380', 'city': 'Chilly-Mazarin', 'country': 'France', 'facility': 'Sanofi-Aventis', 'geoPoint': {'lat': 48.71489, 'lon': 2.31638}}], 'overallOfficials': [{'name': 'Clinical Sciences and Operations', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sanofi'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': "Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sanofi', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}