NCT05208281

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

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 32 (estimated)
• Sponsor: AO GENERIUM · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed description

GNR-055 is intended for ERT in patient with Mucopolysaccharidosis type II (MPS II), or Hunter syndrome. MPS II is a recessive X-linked inheritance lysosomal storage disease, which is characterized by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (ID2S), caused by a mutation in the ID2S gene. Enzyme deficiency leads to the accumulation of Glycosaminoglycans (GAG) (mainly of heparan and dermatan sulfates) in lysosomes of almost all types of cells of various tissues and organs. The disease is manifested by growth retardation, damage of many organs and systems, severe deformations of bones and joints, gross facial features, pathology of the respiratory and cardiovascular systems, damage to parenchymal organs (hepatosplenomegaly), and hearing impairment. A severe form of the disease occurs with the involvement of the nervous system in the pathological process, including mental retardation, behavior anomalies, and impaired motor function. GNR-055 is a recombinant modified ID2S capable to penetrate the blood-brain barrier and thus expected to prevent neurodegenerative consequences and the cognitive deficit and to attain a significant improvement in the life quality and expectancy of patients with MPS II. Study IDB-MPS-II-III is a multicenter, open-label, multi-cohort study to assess safety, PK and PD, and efficacy of GNR-055 in patients of different age groups with MPS II (Hunter syndrome).

Conditions

• Mucopolysaccharidosis Type II
• Metabolic Diseases

Interventions

Type	Name	Description
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

Timeline

• Start date: 2021-11-30
• Primary completion: 2026-06-01
• Completion: 2028-03-01
• First posted: 2022-01-26
• Last updated: 2025-07-30

Locations

5 sites across 1 country: Russia