Ignite Creation Date:
2025-12-25 @ 2:47 AM
Ignite Modification Date:
2025-12-26 @ 1:28 AM
Study NCT ID:
NCT07251933
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-11-26
First Post:
2025-11-19
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D054179', 'term': 'Angioedemas, Hereditary'}], 'ancestors': [{'id': 'D000799', 'term': 'Angioedema'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000081208', 'term': 'Hereditary Complement Deficiency Diseases'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D014581', 'term': 'Urticaria'}, {'id': 'D017445', 'term': 'Skin Diseases, Vascular'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D006969', 'term': 'Hypersensitivity, Immediate'}, {'id': 'D006967', 'term': 'Hypersensitivity'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-01-31', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2026-03-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-19', 'studyFirstSubmitDate': '2025-11-19', 'studyFirstSubmitQcDate': '2025-11-19', 'lastUpdatePostDateStruct': {'date': '2025-11-26', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-11-26', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2026-03-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants who are Free of HAE Attacks', 'timeFrame': 'Up to 13 months', 'description': 'Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).'}], 'secondaryOutcomes': [{'measure': 'HAE Attack Rate of Participants', 'timeFrame': 'Up to 13 months'}, {'measure': 'HAE Attack-free Rate of Participants Relative to Prior Treatment', 'timeFrame': 'Up to 13 months'}, {'measure': 'Association Between Covariates of Interest and Attack Free Rate', 'timeFrame': 'Up to 13 months', 'description': 'Association between covariates of interest (e.g., age, sex, family history) and attack-free rate will be determined. Associations between attack free rate and covariates of interest will be modelled, using generalized linear models.'}, {'measure': 'Number of Participants With HAE Attack Based on Attack Severity', 'timeFrame': 'Up to 13 months', 'description': 'Number of participants with HAE attack based on attack severity (mild, moderate, severe, unknown) will be reported.'}, {'measure': 'Number of Participants With HAE Attack Based on Location', 'timeFrame': 'Up to 13 months', 'description': 'Number of participants with HAE attack based on location (e.g. extremities, abdominal, facial, laryngeal or genital) will be reported.'}, {'measure': 'Number of Participants with Treatment History Before use of Lanadelumab', 'timeFrame': 'Up to 13 months', 'description': 'Number of participants with treatment history (type of treatment, duration of treatment, reason for start/ discontinuation/ switch) before use of lanadelumab will be reported.'}, {'measure': 'Average Duration of Lanadelumab Treatment', 'timeFrame': 'Up to 13 months', 'description': 'Average duration of lanadelumab treatment will be reported.'}, {'measure': 'Number of Participants Characterized Based on Lanadelumab Treatment Patterns', 'timeFrame': 'Up to 13 months', 'description': 'Number of participants based on lanadelumab treatment patterns (e.g. setting of administration, interval of administration changes/dose modifications, reasons for initiation/discontinuation and modifications) will be reported.'}, {'measure': 'Number of Participants With Treatments Received Following Lanadelumab Discontinuation', 'timeFrame': 'Up to 13 months', 'description': 'Number of participants with treatments (e.g. type of treatment, reason for selection) received following lanadelumab discontinuation will be evaluated.'}, {'measure': 'Number of Participants With Healthcare Resource Utilization (HRU) Outcomes', 'timeFrame': 'Up to 13 months', 'description': 'HRU outcomes will include number of healthcare professional (HCP) visits, number of emergency department (ED) visits, number of inpatient hospitalizations, number of participants with admission to and days spent in intensive care unit related to HAE-C1INH, and number of school days missed will be reported.'}, {'measure': 'Duration of Hospital Stay in Days Related to HAE-C1INH Hospitalizations', 'timeFrame': 'Up to 13 months', 'description': 'Length of hospital stay in days related to HAE-C1INH hospitalizations will be reported.'}, {'measure': 'Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Up to 13 months'}, {'measure': 'Percentage of Participants With Injection Site Reactions', 'timeFrame': 'Up to 13 months', 'description': 'Percentage of participants with injection site reactions will be reported.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Drug Therapy'], 'conditions': ['Hereditary Angioedema (HAE)']}, 'descriptionModule': {'briefSummary': "HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment.\n\nThe main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks.\n\nOther goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition.\n\nThe study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Participant is aged 2 to less than (\\<) 12 years at the time of lanadelumab initiation and is expected to have at least 6 months of follow-up information before turning 12.\n2. Participant has physician-confirmed diagnosis of HAE-C1INH.\n3. Participant initiated LTP with lanadelumab during the eligibility period.\n4. Signed consent/assent (where required by local regulations).\n5. Participant's medical record contains documentation of HAE attacks in the pre-index period and after lanadelumab initiation.\n\nExclusion Criteria:\n\n1. Participant was enrolled in a therapeutic investigational drug (lanadelumab or other drug) or device trial at index date.\n2. Participant with no documented HAE attacks in the 12 months prior to index date."}, 'identificationModule': {'nctId': 'NCT07251933', 'briefTitle': 'A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'A Multi-national Real-world Outcomes and Treatment Patterns Study of Lanadelumab (Takhzyro) in Paediatric Patients With Hereditary Angioedema (TAHORA)', 'orgStudyIdInfo': {'id': 'TAK-743-4038'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Participants with HAE-C1INH', 'description': 'Paediatric participants with HAE-C1INH who initiated long-term prophylaxis (LTP) treatment with lanadelumab within a routine clinical setting will be included.', 'interventionNames': ['Other: No intervention']}], 'interventions': [{'name': 'No intervention', 'type': 'OTHER', 'description': 'This is a non-interventional study.', 'armGroupLabels': ['Participants with HAE-C1INH']}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Takeda Contact', 'role': 'CONTACT', 'email': 'medinfoUS@takeda.com', 'phone': '+1-877-825-3327'}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/ourmember/takeda/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'ipdSharing': 'YES', 'description': "Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.", 'accessCriteria': 'IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Takeda', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}