drugset
drugset / Trial / NCT05208281

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

NCT05208281 ↗

Phase 2/3 Recruiting 32 enrolled AO GENERIUM
Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start
2021-11-30
Primary completion
2026-06
Completion
2028-03

Drugs

EvaluationDrugModalityDoseRoute
Subject GNR-055 Protein / enzyme biologic 1 mg/kg Intravenous
Subject GNR-055 Protein / enzyme biologic 2 mg/kg Intravenous
Subject GNR-055 Protein / enzyme biologic 3 mg/kg Intravenous

Indications

mucopolysaccharidosis type 2