RecruitingPhase 2Phase 3NCT05208281

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

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

Sponsor

AO GENERIUM

Enrollment

32 participants

Start Date

Nov 30, 2021

Study Type

INTERVENTIONAL

Conditions

Metabolic Diseases Mucopolysaccharidosis Type II

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.

Eligibility

Sex: MALE

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called GNR-055 1.0-2.0-3.0 mg/kg, a drug called GNR-055 2.0 mg/kg, and others for people with metabolic diseases and mucopolysaccharidosis type ii. The study is currently recruiting participants at 5 locations. People eligible for this study include men.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGGNR-055 1.0-2.0-3.0 mg/kg

Weekly IV infusion (lyophilized powder) 1.0-2.0-3.0 mg/kg

DRUGGNR-055 2.0 mg/kg

Weekly IV infusion (lyophilized powder) 2.0 mg/kg

DRUGGNR-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

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NCT05208281

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