Ignite Creation Date:
2025-12-24 @ 6:42 PM
Ignite Modification Date:
2025-12-29 @ 10:02 PM
Study NCT ID:
NCT07290257
Status:
RECRUITING
Last Update Posted:
2025-12-18
First Post:
2025-09-17
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016738', 'term': 'Alagille Syndrome'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D002779', 'term': 'Cholestasis'}, {'id': 'D011537', 'term': 'Pruritus'}], 'ancestors': [{'id': 'D002780', 'term': 'Cholestasis, Intrahepatic'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012877', 'term': 'Skin Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000722912', 'term': 'maralixibat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'The study will enroll a primary cohort with a minimum of 45 previously untreated (treatment naïve) patients with ALGS including at least 5 participants \\<1 year of age, 30 participants between 1 and 5 years of age, and 10 participants \\>5 years of age. A supplemental cohort of participants who have previously received Livmarli will also be enrolled. There is no enrollment limit for supplementary cohort. For admin purposes, the number has been set to 55 participants.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-09-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2030-12-22', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-04', 'studyFirstSubmitDate': '2025-09-17', 'studyFirstSubmitQcDate': '2025-12-04', 'lastUpdatePostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2030-09-22', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Adverse Events (AEs)', 'timeFrame': 'Up to 7 days after the last dose of Livmarli.', 'description': 'Number and proportion of participants with AEs.'}, {'measure': 'Liver function tests (LFTs) - measuring enzyme activity or concentration', 'timeFrame': 'Once every 3-4 months, for 5 years.', 'description': 'Change in LFTs from Baseline: Total and direct bilirubin, aminotransferases, GGT, and alkaline phosphatase.'}, {'measure': 'Fat-Soluble Vitamins (FSV) Concentration', 'timeFrame': 'Once every 3-4 months, for 5 years.', 'description': 'Change in FSV levels (vitamins A, D, and E) from Baseline.'}, {'measure': 'International Normalized Ratio (INR)', 'timeFrame': 'Once every 3-4 months, for 5 years.', 'description': 'Change in INR level from Baseline.'}, {'measure': 'Long-Term Clinical Outcomes', 'timeFrame': 'Once every 3-4 months, for 5 years.', 'description': 'Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['ALGS', 'Liver Diseases', 'Alagille Syndrome', 'Livmarli', 'Maralixibat', 'Cholestasis', 'Pruritus', 'Itching', 'IBAT inhibitor'], 'conditions': ['Alagille Syndrome']}, 'descriptionModule': {'briefSummary': 'The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.', 'detailedDescription': 'This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with Alagille syndrome (ALGS) who are treated with Livmarli for cholestatic pruritus.\n\nParticipants will be treated according to standard of care for the duration of the study and Schedule of Assessments.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Informed consent and assent (as applicable)\n* ≥2 months of age at Day 1\n* A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis\n* For the primary cohort, prescribed Livmarli at time of study entry\n* For the supplemental cohort, prescribed Livmarli prior to study entry\n\nExclusion Criteria:\n\n* History of Liver Transplant\n* Any contraindications against Livmarli (as per SmPC)\n* Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study\n* Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)\n* Baseline data before start of treatment of Livmarli are unavailable (\\<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter'}, 'identificationModule': {'nctId': 'NCT07290257', 'briefTitle': 'Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mirum Pharmaceuticals, Inc.'}, 'officialTitle': 'Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)', 'orgStudyIdInfo': {'id': 'MRX-803'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Patients with Alagile Syndrome treated with Livmarli', 'description': 'Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli.', 'interventionNames': ['Drug: Livmarli Oral Product']}], 'interventions': [{'name': 'Livmarli Oral Product', 'type': 'DRUG', 'otherNames': ['Maralixibat'], 'description': 'Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.', 'armGroupLabels': ['Patients with Alagile Syndrome treated with Livmarli']}]}, 'contactsLocationsModule': {'locations': [{'zip': '1200', 'city': 'Brussels', 'status': 'NOT_YET_RECRUITING', 'country': 'Belgium', 'contacts': [{'name': 'Stephenne Xavier', 'role': 'CONTACT', 'email': 'xavier.stephenne@uclouvain.be', 'phone': '+32 2 764 13 77'}], 'facility': 'Cliniques Universitaires Saint Luc (UCLouvain)', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'zip': '9000', 'city': 'Ghent', 'status': 'NOT_YET_RECRUITING', 'country': 'Belgium', 'contacts': [{'name': 'Ruth De Bruyne', 'role': 'CONTACT', 'email': 'ruth.debruyne@ugent.be', 'phone': '+32 9 332 39 66'}], 'facility': 'University Hospital Gent (UZ Gent)', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'zip': '69500', 'city': 'Bron', 'state': 'Auvergne-Rhône-Alpes', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Mathias Ruiz', 'role': 'CONTACT', 'email': 'mathias.ruiz@chu-lyon.fr', 'phone': '+33 472 35 70 50'}], 'facility': 'Hospices Civils de Lyon - Hopital Femme Mère Enfant', 'geoPoint': {'lat': 45.73865, 'lon': 4.91303}}, {'zip': '31049', 'city': 'Toulouse', 'state': 'Occitanie', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Nolwenn Laborde', 'role': 'CONTACT', 'email': 'laborde.n@chu-toulouse.fr', 'phone': '+33.(0)5 34558566'}], 'facility': 'CHU de Toulouse - Hôpital des Enfants', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'zip': '94270', 'city': 'Le Kremlin-Bicêtre', 'state': 'Île-de-France Region', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Emmanuel Gonzales', 'role': 'CONTACT', 'email': 'emmanuel.gonzales@aphp.fr', 'phone': '+33 1 45 21 37 88'}], 'facility': 'Bicetre University Hospital', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'zip': '13353', 'city': 'Berlin', 'status': 'NOT_YET_RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Philip Bufler', 'role': 'CONTACT', 'email': 'philip.bufler@charite.de'}], 'facility': 'Charite Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '45149', 'city': 'Essen', 'status': 'NOT_YET_RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Elke Lainka', 'role': 'CONTACT', 'email': 'Elke.Lainka@uk-essen.de', 'phone': '+49 (0)201 723 3360'}], 'facility': "Children's University Hospital Essen", 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'zip': '20246', 'city': 'Hamburg', 'status': 'NOT_YET_RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Sebastian Schulz-Jürgensen', 'role': 'CONTACT', 'email': 's.schulz-juergensen@uke.de', 'phone': '+49 40 7410 52702'}], 'facility': 'University Hospital Hamburg-Eppendorf', 'geoPoint': {'lat': 53.55073, 'lon': 9.99302}}, {'zip': '24126', 'city': 'Bergamo', 'state': 'Lombardy', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': "Lorenzo D'Antiga", 'role': 'CONTACT', 'email': 'ldantiga@asst-pg23.it'}], 'facility': 'AO Ospedale PAPA GIOVANNI XXIII', 'geoPoint': {'lat': 45.69601, 'lon': 9.66721}}, {'zip': '90127', 'city': 'Palermo', 'state': 'Sicily', 'status': 'NOT_YET_RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Giusy Ranucci', 'role': 'CONTACT', 'email': 'granucci@ismett.edu'}], 'facility': 'Istituto mediterraneo trapianti - ISMETT', 'geoPoint': {'lat': 38.1166, 'lon': 13.3636}}, {'zip': '9713GZ', 'city': 'Groningen', 'status': 'NOT_YET_RECRUITING', 'country': 'Netherlands', 'contacts': [{'name': 'Henkjan Verkade', 'role': 'CONTACT', 'email': 'h.j.verkade@umcg.nl', 'phone': '+31 50 3614147'}], 'facility': 'University Medical Center Groningen (UMCG)', 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}, {'zip': '08035', 'city': 'Barcelona', 'state': 'Catalonia', 'status': 'RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Jesus Quintero', 'role': 'CONTACT', 'email': 'jesus.quintero@vallhebron.cat'}], 'facility': "Hospital Universitairo Vall D'Hebron", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28046', 'city': 'Madrid', 'status': 'RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Esteban Frauca Remacha', 'role': 'CONTACT', 'email': 'esteban.frauca@salud.madrid.org'}], 'facility': 'Hospital Universitario La Paz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}], 'centralContacts': [{'name': 'Clinical Trials Mirum', 'role': 'CONTACT', 'email': 'clinicaltrials@mirumpharma.com', 'phone': '+16506674085'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mirum Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}