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A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Sponsor: AO GENERIUM
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.
Official title: Multicenter, Open-Label, Multi-cohort Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Drug Product GNR 055 (JSC "GENERIUM", Russia) in Patients With Mucopolysaccharidosis Type II
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
32
Start Date
2021-11-30
Completion Date
2028-03
Last Updated
2025-07-30
Healthy Volunteers
No
Interventions
GNR-055 1.0-2.0-3.0 mg/kg
Weekly IV infusion (lyophilized powder) 1.0-2.0-3.0 mg/kg
GNR-055 2.0 mg/kg
Weekly IV infusion (lyophilized powder) 2.0 mg/kg
GNR-055 3.0 mg/kg
Weekly IV infusion (lyophilized powder) 3.0 mg/kg
Locations (5)
Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Academy of Sciences (Research Institute of Pediatrics and Child Health Protection of the Central Clinical Hospital of the Russian Academy of Sciences)
Moscow, Russia
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation
Saint Petersburg, Russia
V.I. Vernadsky Crimean Federal University
Simferopol, Russia
State Budgetary Healthcare Institution Republican Medical Genetic Center
Ufa, Russia
State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital
Yekaterinburg, Russia