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Study identifier: NCT05208281 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Mucopolysaccharidosis Type II · Metabolic Diseases  ·  Sponsor: AO GENERIUM

PhasePhase 2/Phase 3
Planned participants32
Who can joinMale, N/A to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Svetlana B. Korotkova, MD, PhD  ·  +7(495) 988 47 94  ·  sbkorotkova@generium.ru

Oksana A. Markova, MD, MSc  ·  +7(495) 988 47 94  ·  oamarkova@generium.ru

Always discuss trial participation with your own doctor first.

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, RussiaNot Yet Recruiting
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian FederationSaint Petersburg, RussiaNot Yet Recruiting
V.I. Vernadsky Crimean Federal UniversitySimferopol, RussiaRecruiting
State Budgetary Healthcare Institution Republican Medical Genetic CenterUfa, RussiaRecruiting
State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical HospitalYekaterinburg, RussiaRecruiting

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Source record: clinicaltrials.gov/study/NCT05208281