Ignite Creation Date:
2025-12-24 @ 5:43 PM
Ignite Modification Date:
2026-01-02 @ 1:55 PM
Study NCT ID:
NCT05453968
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-10
First Post:
2022-06-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Berotralstat Treatment in Children With Hereditary Angioedema
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['North Macedonia'], 'submissionTracking': {'submissionInfos': [{'releaseDate': '2025-12-19'}], 'estimatedResultsFirstSubmitDate': '2025-12-19'}}, '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000706836', 'term': 'berotralstat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'This is a sequential, three-part, open-label study. Minimum participation in this study is expected to be 24 weeks in the SOC treatment period through Part 1 of the study and up to an additional 132 weeks in Parts 2 and 3.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2022-10-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'dispFirstSubmitDate': '2025-09-08', 'completionDateStruct': {'date': '2027-02', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-08', 'studyFirstSubmitDate': '2022-06-30', 'studyFirstSubmitQcDate': '2022-07-08', 'dispFirstPostDateStruct': {'date': '2024-10-15', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-09-10', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-07-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-09-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Acceptability/palatability of berotralstat oral granules using a self-reported taste rating scale designed with images centered on taste', 'timeFrame': 'Time of first dose (Day 1)', 'description': 'TASTY; 7-point scale \\[0 "worst" to 6 "best"\\]'}], 'primaryOutcomes': [{'measure': 'Pharmacokinetics: Cmax', 'timeFrame': 'Predose and multiple timepoints up to 24 hours postdose', 'description': 'Maximum plasma concentration of berotralstat'}, {'measure': 'Pharmacokinetics: AUC0-tau', 'timeFrame': 'Predose and multiple timepoints up to 24 hours postdose', 'description': 'Area under the plasma concentration berotralstat time curve from time zero to the end of dosing (tau)'}, {'measure': 'Pharmacokinetics: CL/F', 'timeFrame': 'Predose and multiple timepoints up to 24 hours postdose', 'description': 'Apparent oral clearance of berotralstat'}], 'secondaryOutcomes': [{'measure': 'Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)', 'timeFrame': 'Over 144 weeks'}, {'measure': 'Frequency and severity of hereditary angioedema (HAE) attacks', 'timeFrame': 'Over 48 weeks'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Berotralstat', 'BCX7353', 'Orladeyo®', 'once a day', 'Hereditary angioedema (HAE)', 'Pediatric', 'oral once a day dosing', 'kallikrein inhibitor'], 'conditions': ['Hereditary Angioedema', 'Pediatric']}, 'referencesModule': {'references': [{'pmid': '40716738', 'type': 'DERIVED', 'citation': 'Bernatoniene J, Bourgoin-Heck M, Cancian M, Yang W, Hagin D, Pagnier A, Stobiecki M, Kinaciyan T, Phillips-Angles E, Gayet S, Bara NA, Hunter J, Mateescu E, DeSpirito M, Johnston D, Long D, Iocca H, Petroni D, Aygoren-Pursun E. Oral berotralstat for hereditary angioedema prophylaxis in patients aged 2 to <12 years: APeX-P interim results. Ann Allergy Asthma Immunol. 2025 Dec;135(6):681-688.e3. doi: 10.1016/j.anai.2025.07.012. Epub 2025 Jul 25.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to \\< 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).', 'detailedDescription': 'This is a single-arm, open-label study designed to evaluate the PK and safety of berotralstat weight-based treatment for the prevention of hereditary angioedema attacks in pediatric participants 2 to \\< 12 years of age. This study will consist of two treatment periods: a 12-week standard-of-care (SOC) treatment period followed by an open-label berotralstat treatment period lasting up to 144 weeks.\n\nParticipants will be enrolled into 4 dose cohorts; participant weight will be used to determine assignment to each cohort with the higher weight cohorts (Cohorts 1 and 2) enrolling first and in parallel. Safety assessments and PK modelling from all available PK data will then be used to confirm the weight bands for sequentially enrolling Cohorts 3 and 4.The effectiveness of berotralstat in this population will be summarized using descriptive statistical methods.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Male and non-pregnant, non-lactating females 2 to \\< 12 years of age\n* Body weight ≥ 12 kg\n* Clinical diagnosis of HAE\n* In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis\n\nExclusion Criteria:\n\n* Concurrent diagnosis of any other type of recurrent angioedema\n* Known family history of sudden cardiac death\n* Creatinine clearance using the modified Schwartz formula of ≤ 30 mL/min/1.73 m2\n* Aspartate aminotransferase or alanine aminotransferase value ≥ 3 × the upper limit of the age-appropriate normal reference range value\n* Clinically significant abnormal ECG including but not limited to, a corrected QT interval calculated using Fridericia's correction \\> 450 msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping\n* Current participation in any other investigational drug study or received another investigational drug within 30 days of enrollment"}, 'identificationModule': {'nctId': 'NCT05453968', 'acronym': 'APeX-P', 'briefTitle': 'Berotralstat Treatment in Children With Hereditary Angioedema', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioCryst Pharmaceuticals'}, 'officialTitle': 'A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age', 'orgStudyIdInfo': {'id': 'BCX7353-304'}, 'secondaryIdInfos': [{'id': '2021-005932-50', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Berotralstat', 'description': 'Berotralstat administered once daily in 4 dose cohorts determined by participant weight. Cohorts 1 and 2 will enroll in parallel. After 4 participants from Cohort 1 and 2, with ≥ 2 subjects from Cohort 2, have reached Week 2, Cohort 3 will open for enrollment. Cohort 4 will open for enrollment, after ≥ 4 subjects in Cohort 3 have reached Week 2. Prior to dosing Cohort 3 and 4, available PK and safety data will be reviewed to confirm it is safe to proceed and the appropriate weight bands for each. BioCryst will notify sites when Cohorts 3 and 4 are open for enrollment.', 'interventionNames': ['Drug: Berotralstat']}], 'interventions': [{'name': 'Berotralstat', 'type': 'DRUG', 'otherNames': ['BCX7353', 'Orladeyo®'], 'description': 'Administered orally once daily at a weight-based dose in up to 4 cohorts', 'armGroupLabels': ['Berotralstat']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Vienna', 'country': 'Austria', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'city': 'Grenoble', 'country': 'France', 'facility': 'Investigative Site #3', 'geoPoint': {'lat': 45.17869, 'lon': 5.71479}}, {'city': 'Marseille', 'country': 'France', 'facility': 'Investigative Site #2', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'city': 'Paris', 'country': 'France', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Berlin', 'country': 'Germany', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Investigative Site #2', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'city': 'Haifa', 'country': 'Israel', 'facility': 'Investigative Site #2', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'city': 'Padua', 'country': 'Italy', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'city': 'Krakow', 'country': 'Poland', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 50.06143, 'lon': 19.93658}}, {'city': 'Sângeorgiu de Mureş', 'country': 'Romania', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 46.5696, 'lon': 24.59453}}, {'city': 'Madrid', 'country': 'Spain', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'city': 'Málaga', 'country': 'Spain', 'facility': 'Investigative Site #2', 'geoPoint': {'lat': 36.72016, 'lon': -4.42034}}, {'city': 'Bristol', 'country': 'United Kingdom', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 51.45523, 'lon': -2.59665}}], 'overallOfficials': [{'name': 'Jolanta Bernatoniene, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Bristol Royal Hospital for Children'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioCryst Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}