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

PHASE2/PHASE3RecruitingINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

November 30, 2021

Primary Completion Date

June 30, 2026

Study Completion Date

March 31, 2028

Conditions
Mucopolysaccharidosis Type IIMetabolic Diseases
Interventions
DRUG

GNR-055 1.0-2.0-3.0 mg/kg

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

DRUG

GNR-055 2.0 mg/kg

Weekly IV infusion (lyophilized powder) 2.0 mg/kg

DRUG

GNR-055 3.0 mg/kg

Weekly IV infusion (lyophilized powder) 3.0 mg/kg

Trial Locations (5)

119333

NOT_YET_RECRUITING

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

194100

NOT_YET_RECRUITING

"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

295007

RECRUITING

V.I. Vernadsky Crimean Federal University, Simferopol

450076

RECRUITING

State Budgetary Healthcare Institution Republican Medical Genetic Center, Ufa

620149

RECRUITING

State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital, Yekaterinburg

All Listed Sponsors
lead

AO GENERIUM

INDUSTRY