Ignite Creation Date:
2026-03-26 @ 3:19 PM
Ignite Modification Date:
2026-03-30 @ 3:51 AM
Study NCT ID:
NCT07446894
Status:
RECRUITING
Last Update Posted:
2026-03-04
First Post:
2026-02-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
MSA-01 in Multiple System Atrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019578', 'term': 'Multiple System Atrophy'}], 'ancestors': [{'id': 'D054969', 'term': 'Primary Dysautonomias'}, {'id': 'D001342', 'term': 'Autonomic Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D000080874', 'term': 'Synucleinopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 140}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2026-02-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-03', 'completionDateStruct': {'date': '2028-03-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-02', 'studyFirstSubmitDate': '2026-02-25', 'studyFirstSubmitQcDate': '2026-02-25', 'lastUpdatePostDateStruct': {'date': '2026-03-04', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-03-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-03-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change from baseline in individual UMSARS Part 2 item scores', 'timeFrame': 'Baseline to Week 52'}, {'measure': 'Change from baseline in individual UMSARS Part 1 item scores', 'timeFrame': 'Baseline to Week 52'}, {'measure': 'Change from baseline in the scores of the four ADL-related items of UMSARS Part 1', 'timeFrame': 'Baseline to Week 52'}, {'measure': 'Change from baseline in the scores of the eight QOL-related items of UMSARS Part 1', 'timeFrame': 'Baseline to Week 52'}, {'measure': 'Change from baseline in the EQ-5D-5L index score', 'timeFrame': 'Baseline to Week 52'}], 'primaryOutcomes': [{'measure': 'Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score', 'timeFrame': 'Baseline to Week 52'}], 'secondaryOutcomes': [{'measure': 'Change from baseline to Week 52 in the Barthel Index', 'timeFrame': 'Baseline to Week 52'}, {'measure': 'Change from baseline to Week 52 in the Scale for the Assessment and Rating of Ataxia (SARA) score', 'timeFrame': 'Baseline to Week 52'}, {'measure': 'Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score', 'timeFrame': 'Baseline to Week 52'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Multiple System Atrophy (MSA)']}, 'referencesModule': {'references': [{'pmid': '37256098', 'type': 'BACKGROUND', 'citation': 'Mitsui J, Matsukawa T, Uemura Y, Kawahara T, Chikada A, Porto KJL, Naruse H, Tanaka M, Ishiura H, Toda T, Kuzuyama H, Hirano M, Wada I, Ga T, Moritoyo T, Takahashi Y, Mizusawa H, Ishikawa K, Yokota T, Kuwabara S, Sawamoto N, Takahashi R, Abe K, Ishihara T, Onodera O, Matsuse D, Yamasaki R, Kira JI, Katsuno M, Hanajima R, Ogata K, Takashima H, Matsushima M, Yabe I, Sasaki H, Tsuji S. High-dose ubiquinol supplementation in multiple-system atrophy: a multicentre, randomised, double-blinded, placebo-controlled phase 2 trial. EClinicalMedicine. 2023 Apr 14;59:101920. doi: 10.1016/j.eclinm.2023.101920. eCollection 2023 May.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate whether MSA-01 slows the progression of multiple system atrophy (MSA) and to assess its safety.\n\nThe primary question is:\n\n• Does MSA-01 slow the progression of motor impairment as measured by UMSARS Part 2 score?\n\nParticipants will be randomly assigned to receive MSA-01 or placebo for 12 months. They will attend regular clinic visits for safety and efficacy assessments and record their medication use and any side effects in a diary.', 'detailedDescription': 'Multiple system atrophy (MSA) is a progressive neurodegenerative disorder with no established disease-modifying treatment. Evidence suggests that coenzyme Q10 (CoQ10) deficiency may contribute to MSA pathophysiology. MSA-01 (ubiquinol), a highly bioavailable form of CoQ10, demonstrated acceptable safety and potential efficacy in a prior phase II trial.\n\nThis is a multicenter, randomized, double-blind, placebo-controlled phase III study evaluating the efficacy and safety of MSA-01 in patients with MSA. Approximately 140 participants will be randomized 1:1 to receive oral MSA-01 or placebo for 52 weeks.\n\nThe primary endpoint is the change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score. Secondary endpoints include additional clinical scales and safety assessments. Efficacy will be analyzed using a mixed-effects model for repeated measures. The study aims to determine whether MSA-01 slows clinical progression compared with placebo while maintaining an acceptable safety profile.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '79 Years', 'minimumAge': '30 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\nAt the time of informed consent\n\n1. Patients diagnosed as 'clinically established' or 'clinically probable' MSA based on the revised MSA diagnostic criteria of the Movement Disorder Society (MDS).\n2. Patients who are able to walk independently or with the use of assistive devices.\n3. Patients who are able to attend outpatient visits at the participating study site.\n\n At the start of study drug administration\n4. Patients who are able to discontinue the use of CoQ10 supplements.\n\nExclusion Criteria:\n\n1. Patients with severe neurological disorders, other progressive movement disorders, or cognitive impairment.\n2. Patients with severe liver disease.\n3. Patients with a known history of hypersensitivity to any component of the investigational drug.\n4. Pregnant women, breastfeeding women, or women who may be pregnant.\n5. Patients who have previously participated in a clinical trial of MSA-01."}, 'identificationModule': {'nctId': 'NCT07446894', 'acronym': 'MSA-01_P3', 'briefTitle': 'MSA-01 in Multiple System Atrophy', 'organization': {'class': 'OTHER', 'fullName': 'Tokyo University'}, 'officialTitle': 'A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Study of MSA-01 in Patients With Multiple System Atrophy', 'orgStudyIdInfo': {'id': '2025024-11DX'}, 'secondaryIdInfos': [{'id': 'jRCT2031250696', 'type': 'REGISTRY', 'domain': 'Japan Registry of Clinical Trials'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'MSA-01', 'interventionNames': ['Drug: MSA-01']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'MSA-01', 'type': 'DRUG', 'description': 'Ubiquinol', 'armGroupLabels': ['MSA-01']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Nagoya', 'state': 'Aichi-ken', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Masahisa Katsuno', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Nagoya University Hospital', 'geoPoint': {'lat': 35.18147, 'lon': 136.90641}}, {'city': 'Chiba', 'state': 'Chiba', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Atsuhiko Sugiyama', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Chiba University Hospital', 'geoPoint': {'lat': 35.6, 'lon': 140.11667}}, {'city': 'Fukuoka', 'state': 'Fukuoka', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Dai Matsuse', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Kyushu University Hospital', 'geoPoint': {'lat': 33.6, 'lon': 130.41667}}, {'city': 'Sapporo', 'state': 'Hokkaido', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Ichiro Yabe', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Hokkaido University Hospital', 'geoPoint': {'lat': 43.06667, 'lon': 141.35}}, {'city': 'Kagoshima', 'state': 'Kagoshima-ken', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Yujiro Higuchi', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Kagoshima University Hospital', 'geoPoint': {'lat': 31.56667, 'lon': 130.55}}, {'city': 'Kyoto', 'state': 'Kyoto', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Hodaka Yamakado', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Kyoto University Hospital', 'geoPoint': {'lat': 35.02107, 'lon': 135.75385}}, {'city': 'Okayama', 'state': 'Okayama-ken', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Hiroyuki Ishiura', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Okayama University Hospital', 'geoPoint': {'lat': 34.65, 'lon': 133.93333}}, {'city': 'Hasuda', 'state': 'Saitama', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Katsuhisa Ogata', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'NHO Higashisaitama National Hospital', 'geoPoint': {'lat': 35.97113, 'lon': 139.6491}}, {'city': 'Bunkyō-Ku', 'state': 'Tokyo', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Kinya Ishikawa', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Institute of Science Tokyo Hospital'}, {'city': 'Bunkyō-Ku', 'state': 'Tokyo', 'status': 'RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Jun Mitsui', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}, {'name': 'Ikue Wada', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'The University of Tokyo Hospital'}, {'city': 'Kodaira', 'state': 'Tokyo', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Yuji Takahashi', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'National Center Hospital, National Center of Neurology and Psychiatry', 'geoPoint': {'lat': 35.72603, 'lon': 139.48508}}, {'city': 'Yonago', 'state': 'Tottori', 'status': 'NOT_YET_RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Ritsuko Hanajima', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}], 'facility': 'Tottori University Hospital', 'geoPoint': {'lat': 35.43333, 'lon': 133.33333}}], 'centralContacts': [{'name': 'Jun Mitsui', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}, {'name': 'Ikue Wada', 'role': 'CONTACT', 'email': 'msa-office@umin.ac.jp', 'phone': '+81-3-5800-9762'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Tokyo University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Nobelpharma', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Project Associate Professor', 'investigatorFullName': 'Jun Mitsui', 'investigatorAffiliation': 'Tokyo University'}}}}