Clinical Trial

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

Recruiting Phase 2/3
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Record status
This record was last updated July 30, 2025 (before its estimated June 2026 completion). Its status may not reflect the trial's current state.
Summary
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2022-01-13; most recent amendment 2025-07-28.
Trial Details
NCT Number NCT05208281
Lead Sponsor AO GENERIUM
Conditions Mucopolysaccharidosis Type II, Metabolic Diseases
Enrollment 32 participants
Start Date 2021-11-30
Primary Completion 2026-06 (estimated)
Study Completion 2028-03 (estimated)
Updated on ClinicalTrials.gov 2025-07-30