Actively Recruiting
Multicenter, Open-Label Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of GNR-055 in Patients With Mucopolysaccharidosis Type II
Led by AO GENERIUM · Updated on 2025-07-30
32
Participants Needed
5
Research Sites
91 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and effectiveness of an investigational drug called GNR-055 in patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. This condition is a genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (ID2S), leading to harmful buildup of certain substances in cells that affects growth, organs, and the nervous system. The study is a phase 2/3, multicenter, open-label trial involving different age groups to better understand how GNR-055 works and its safety profile. GNR-055 is a modified enzyme replacement therapy designed to cross the blood-brain barrier, potentially preventing neurological damage and improving quality of life for patients with MPS II. Participants receive weekly intravenous infusions of GNR-055 at doses ranging from 1.0 to 3.0 mg/kg, depending on their study group. The study includes multiple cohorts, with adult and pediatric patients receiving specific dosing regimens over the trial period. During the study, participants will undergo various assessments including monitoring of adverse events, urine and serum levels of glycosaminoglycans (GAG), cerebrospinal fluid analysis, joint motion measurements, MRI scans of liver, spleen, and brain, heart and lung function tests, neurocognitive evaluations, and biomarker analysis. These evaluations occur at baseline and multiple follow-up visits up to week 56. The study aims to gather detailed data on the drug's impact on disease symptoms, safety, and biological markers to inform future treatment options.
CONDITIONS
Brief 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)
- Naïve patients or those who 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
- History of hematopoietic stem cell transplantation or bone marrow transplantation
- Implanted or external non-removable metal devices, cardiac pacemaker, or other objects that may interfere with MRI safety or operation
- Concomitant diseases or conditions that may risk patient safety or affect safety data analysis during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive weekly intravenous infusions of GNR-055 at doses of 1.0, 2.0, or 3.0 mg/kg to evaluate safety, pharmacokinetics, pharmacodynamics, and efficacy.
Weekly visits for up to 52 weeks
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
O
Oksana A. Markova, MD, MSc
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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