A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II

JR-141-101

The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below, * to collect the safety information of JR-141 * to evaluate the plasma pharmacokinetics of JR-141 * to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms

2017-03-30

2017-10-04

2017-10-04

Completed

Early phase I

14

Inclusion Criteria: * Patients aged 6 years or older at the time of informed consent. * Patients diagnosed with MPS II. * Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141. Exclusion Criteria: * Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation. * Patients in whom lumbar puncture cannot be performed. * Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study. * Patients who have received other investigational products within 4 months before enrollment in the study.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 14, 'type': 'ACTUAL'}}

2022-11-14