Ignite Creation Date:
2025-12-24 @ 4:42 PM
Ignite Modification Date:
2025-12-30 @ 5:25 AM
Study NCT ID:
NCT05819866
Status:
RECRUITING
Last Update Posted:
2025-11-17
First Post:
2023-03-23
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C000720427', 'term': 'leriglitazone'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-07-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-05', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-14', 'studyFirstSubmitDate': '2023-03-23', 'studyFirstSubmitQcDate': '2023-04-05', 'lastUpdatePostDateStruct': {'date': '2025-11-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-04-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-05', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.', 'timeFrame': 'Interim analysis 1 (at 18 months of treatment)', 'description': 'The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.'}, {'measure': 'The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.', 'timeFrame': 'Interim analysis 2 (at 27 months of treatment)', 'description': 'The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.'}, {'measure': 'The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.', 'timeFrame': 'Final analysis (at 36 months of treatment)', 'description': 'The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.'}], 'secondaryOutcomes': [{'measure': 'Change from Baseline in Loes Score.', 'timeFrame': 'Interim analysis 1 (at 18 months of treatment)', 'description': 'Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome'}, {'measure': 'Change from Baseline in Loes Score', 'timeFrame': 'Interim analysis 2 (at 27 months of treatment)', 'description': 'Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome'}, {'measure': 'Change from Baseline in Loes Score', 'timeFrame': 'Final analysis (at 36 months of treatment)', 'description': 'Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cerebral Adrenoleukodystrophy (cALD)']}, 'descriptionModule': {'briefSummary': 'A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Subject is male and aged ≥18 years.\n* Subject has progressive cALD, defined as GdE+ brain lesions.\n* Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.\n* Subject has a Loes score ≥0.5 and ≤12 at Screening.\n* Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease\n* Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening.\n\nKey Exclusion Criteria:\n\n* Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).\n* Subject has known type 1 or type 2 diabetes.\n* Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione.\n* Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening.\n* Subject with current participation in another interventional clinical study or within 1 month prior to Screening.\n* Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.'}, 'identificationModule': {'nctId': 'NCT05819866', 'acronym': 'CALYX', 'briefTitle': 'A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Minoryx Therapeutics, S.L.'}, 'officialTitle': 'A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy', 'orgStudyIdInfo': {'id': 'MT-3-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Leriglitazone', 'description': 'Leriglitazone Treatment', 'interventionNames': ['Drug: Leriglitazone']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Leriglitazone', 'type': 'DRUG', 'description': 'Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml', 'armGroupLabels': ['Leriglitazone']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo will match the study drug visually and by taste', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94304-5978', 'city': 'Palo Alto', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jacinda Sampson', 'role': 'CONTACT', 'email': 'jacindas@stanford.edu', 'phone': '650-723-0993'}], 'facility': 'Stanford University Medical Center', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '32608', 'city': 'Gainesville', 'state': 'Florida', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Neuro Medicine Hospital / UF Health', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Eric Mallack', 'role': 'CONTACT', 'email': 'Mallack@kennedykrieger.org', 'phone': '443-923-2750'}], 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Reza Seyedsadjadi', 'role': 'CONTACT', 'email': 'rseyedsadjadi@mgh.harvard.edu', 'phone': '617-726-5175'}], 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55455', 'city': 'Minneota', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Troy Lund', 'role': 'CONTACT', 'email': 'lundx072@umn.edu', 'phone': '612-625-4185'}], 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.55885, 'lon': -95.98559}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Health University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'city': 'Buenos Aires', 'status': 'RECRUITING', 'country': 'Argentina', 'contacts': [{'name': 'Hernan Amartino', 'role': 'CONTACT', 'email': 'carolina.acevedo@resolutioncrs.com', 'phone': '+542304387458'}], 'facility': 'Hospital Austral', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'São Paulo', 'status': 'RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Paulo Sgobbi', 'role': 'CONTACT', 'email': 'pvsgobbi@gmail.com', 'phone': '+55(11)913323742'}], 'facility': 'Federal University of Sao Paulo', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'zip': '75013', 'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Fanny Mochel', 'role': 'CONTACT', 'email': 'fanny.mochel@icm-institute.org', 'phone': '+ 33 (0)1 57 27 41 23'}], 'facility': 'ICM La Pitie Salpetriere', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '04103', 'city': 'Leipzig', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Caroline Bergner', 'role': 'CONTACT', 'email': 'Caroline.Bergner@medizin.uni-leipzig.de', 'phone': '+49 341 97 11747'}], 'facility': 'Klinik und Poliklinik für Neurologie-Leipzig', 'geoPoint': {'lat': 51.33962, 'lon': 12.37129}}, {'zip': '110060', 'city': 'New Delhi', 'status': 'RECRUITING', 'country': 'India', 'contacts': [{'name': 'C.S Agrawal', 'role': 'CONTACT', 'email': 'csyvaa@gmail.com', 'phone': '+91 11 4225 1099'}], 'facility': 'Sir Ganga Ram Hospital', 'geoPoint': {'lat': 28.62137, 'lon': 77.2148}}, {'zip': '28041', 'city': 'Madrid', 'status': 'RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Montserrat Morales', 'role': 'CONTACT', 'email': 'montserrat.morales@salud.madrid.org', 'phone': '630 742 050'}], 'facility': 'Hospital 12 de Octubre', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': 'WC1N 3AX', 'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Robin Lachman', 'role': 'CONTACT', 'email': 'r.lachmann@nhs.net', 'phone': '+442034484778'}], 'facility': 'National Hospital for Neurology and Neurosurgery', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Minoryx Therapeutics, S.L.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}