Actively Recruiting
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Led by AO GENERIUM · Updated on 2025-07-30
32
Participants Needed
5
Research Sites
326 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent
- Verified diagnosis of Mucopolysaccharidosis Type II (Hunter syndrome)
- Patients who are treatment-naïve or have received standard enzyme replacement therapy with idursulfase products
- No contraindications for lumbar puncture as judged by the investigator
- Willingness and ability to follow study procedures
You will not qualify if you...
- Clinically significant hypersensitivity to iduronate-2-sulfatase or any component of the drug product
- History of hematopoietic stem cell transplantation or bone marrow transplantation
- Presence of implanted or external non-removable metal devices, cardiac pacemaker, or other magnetic field-sensitive objects that pose safety risks or interfere with MRI
- Other diseases or conditions that could put patient safety at risk or affect safety data analysis as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
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, 119333
Not Yet Recruiting
2
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, 194100
Not Yet Recruiting
3
V.I. Vernadsky Crimean Federal University
Simferopol, Russia, 295007
Actively Recruiting
4
State Budgetary Healthcare Institution Republican Medical Genetic Center
Ufa, Russia, 450076
Actively Recruiting
5
State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital
Yekaterinburg, Russia, 620149
Actively Recruiting
Research Team
S
Svetlana B. Korotkova, MD, PhD
CONTACT
O
Oksana A. Markova, MD, MSc
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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