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

NCT05208281 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2025-07-30

No results posted yet for this study

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.

Conditions

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

Sponsors & Collaborators

  • AO GENERIUM

    lead INDUSTRY

Principal Investigators

  • Oksana A. Markova, MD, MSc · AO GENERIUM

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-30
Primary Completion
2026-06-30
Completion
2028-03-31

Countries

  • Russia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05208281 on ClinicalTrials.gov