Phase 3, Double-blind, Randomized, Active-controlled (Part 1) and Open-labeled, Historical Placebo Controlled (Part 2) Study to Evaluate the Efficacy of Hunterase (Idursulfase-beta) in Hunter Syndrome (Mucopolysaccharidosis II) Patients

GC1111_P3

The objective of this study is to evaluate the efficacy of GC1111 in Hunter Syndrome Patients

2017-03-09

2022-02-15

2022-02-15

Completed

Early phase I

32

Inclusion Criteria: * Patients diagnosed with Hunter syndrome * Male at the age of ≥ 5 * Adequate abilities (including 6-MWT) to participate in this study in the opinion of the investigator. * Voluntarily signed written informed consent to participation in this study * Consent to contraception Exclusion Criteria: * Prior treatment with iduronate-2-sulfatase ERT * History of bronchotomy, bone marrow trasplanation, or cord blood transplanation. * Known hypersensitivity reactions to any of the components of the invetigational product * Prior or planned administration of other investigational products within 30 days before treatment with the investigational product in this study or duirng this study. * Unable to perform 6-MWT. * Female

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 32, 'type': 'ACTUAL'}}

2024-04-16