Ignite Creation Date:
2025-12-25 @ 1:19 AM
Ignite Modification Date:
2026-02-22 @ 11:11 AM
Study NCT ID:
NCT03128593
Status:
COMPLETED
Last Update Posted:
2022-11-14
First Post:
2017-04-10
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016532', 'term': 'Mucopolysaccharidosis II'}], 'ancestors': [{'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-03-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-11', 'completionDateStruct': {'date': '2017-10-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-11-10', 'studyFirstSubmitDate': '2017-04-10', 'studyFirstSubmitQcDate': '2017-04-20', 'lastUpdatePostDateStruct': {'date': '2022-11-14', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-04-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-10-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of participants with Adverse Events', 'timeFrame': '4 weeks', 'description': '* Adverse events\n* Laboratory tests\n* Vital signs\n* 12-lead electrocardiogram\n* Antibody\n* Infusion associated reaction'}], 'secondaryOutcomes': [{'measure': 'Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]', 'timeFrame': '4 weeks', 'description': 'Plasma concentration of JR-141'}, {'measure': 'Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]', 'timeFrame': '4 weeks', 'description': 'Plasma concentration of JR-141'}, {'measure': 'Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)', 'timeFrame': '4 weeks'}, {'measure': 'HS/DS in CSF', 'timeFrame': '4 weeks', 'description': 'JR-141 concentration in CSF'}, {'measure': 'Urinary total GAG', 'timeFrame': '4 weeks'}, {'measure': 'Liver and spleen volumes', 'timeFrame': '4 weeks'}, {'measure': 'Cardiac function', 'timeFrame': '4 weeks'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Mucopolysaccharidosis II']}, 'referencesModule': {'references': [{'pmid': '30595526', 'type': 'DERIVED', 'citation': 'Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y. Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2019 Feb 6;27(2):456-464. doi: 10.1016/j.ymthe.2018.12.005. Epub 2018 Dec 8.'}], 'seeAlsoLinks': [{'url': 'https://pubmed.ncbi.nlm.nih.gov/30595526/', 'label': 'Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,\n\n* to collect the safety information of JR-141\n* to evaluate the plasma pharmacokinetics of JR-141\n* to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients aged 6 years or older at the time of informed consent.\n* Patients diagnosed with MPS II.\n* Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.\n\nExclusion Criteria:\n\n* Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.\n* Patients in whom lumbar puncture cannot be performed.\n* Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.\n* Patients who have received other investigational products within 4 months before enrollment in the study.'}, 'identificationModule': {'nctId': 'NCT03128593', 'briefTitle': 'A Study of JR-141 in Patients With Mucopolysaccharidosis Type II', 'organization': {'class': 'INDUSTRY', 'fullName': 'JCR Pharmaceuticals Co., Ltd.'}, 'officialTitle': 'A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II', 'orgStudyIdInfo': {'id': 'JR-141-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental: JR-141', 'interventionNames': ['Drug: JR-141']}], 'interventions': [{'name': 'JR-141', 'type': 'DRUG', 'description': 'IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week', 'armGroupLabels': ['Experimental: JR-141']}]}, 'contactsLocationsModule': {'locations': [{'zip': '501-1194', 'city': 'Gifu', 'country': 'Japan', 'facility': 'Gifu Clinical site', 'geoPoint': {'lat': 35.42291, 'lon': 136.76039}}, {'zip': '830-0011', 'city': 'Kurume', 'country': 'Japan', 'facility': 'Fukuoka Clinical site', 'geoPoint': {'lat': 33.31667, 'lon': 130.51667}}, {'zip': '105-8471', 'city': 'Minato', 'country': 'Japan', 'facility': 'Tokyo clinical site1', 'geoPoint': {'lat': 34.2152, 'lon': 135.1501}}, {'zip': '545-8586', 'city': 'Osaka', 'country': 'Japan', 'facility': 'Osaka Clinical site1', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'zip': '330-8777', 'city': 'Saitama', 'country': 'Japan', 'facility': 'Saitama Clinical site', 'geoPoint': {'lat': 35.90807, 'lon': 139.65657}}, {'zip': '157-8535', 'city': 'Setagaya City', 'country': 'Japan', 'facility': 'Tokyo clinical site2', 'geoPoint': {'lat': 35.64188, 'lon': 139.64715}}, {'zip': '157-8535', 'city': 'Suita', 'country': 'Japan', 'facility': 'Osaka Clinical site2', 'geoPoint': {'lat': 34.76143, 'lon': 135.51567}}, {'zip': '683-8504', 'city': 'Yonago', 'country': 'Japan', 'facility': 'Tottori Clinical site', 'geoPoint': {'lat': 35.43333, 'lon': 133.33333}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'JCR Pharmaceuticals Co., Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}