Evaluating GNR-055 for treating 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
This study is testing a new treatment called GNR-055 to see if it can improve the health and quality of life for people with Mucopolysaccharidosis Type II, also known as Hunter syndrome.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 32 (estimated) |
| Sex | Male |
| Sponsor | Ao Generium Industry-sponsored |
| Locations | 5 sites (Moscow and 4 other locations) |
| Trial ID | NCT05208281 on ClinicalTrials.gov |
What this trial studies
This multi-cohort study aims to assess the safety, pharmacokinetics, pharmacodynamics, and efficacy of GNR-055 in patients with Mucopolysaccharidosis Type II (Hunter syndrome) across different age groups. GNR-055 is a recombinant enzyme designed to penetrate the blood-brain barrier, potentially preventing neurodegenerative effects and cognitive deficits associated with the disease. The study will involve administering varying doses of GNR-055 to evaluate its impact on the quality of life and overall health of participants. It is an open-label study, meaning both the researchers and participants will know the treatment being administered.
Who should consider this trial
Good fit: Ideal candidates include patients diagnosed with MPS II who are either treatment-naïve or have previously received standard enzyme replacement therapy.
Not a fit: Patients with a history of hematopoietic stem cell transplantation or those with contraindications for lumbar puncture may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life and life expectancy for patients with MPS II.
How similar studies have performed: While this approach is novel in its specific application, similar studies targeting enzyme replacement therapies for lysosomal storage diseases have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Signed inform consent; * Verified diagnosis of MPS II (Hunter syndrome); * Naïve patients or patients who have received standard ERT whit idursulfase products; * No contraindications for lumbar puncture as judged by the Investigator; * Willingness and ability to follow study procedures. Exclusion Criteria: * Clinically pronounced hypersensitivity to ID2S or any other component of the drug product; * History of hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation; * Implanted or external non-removable metal devices, a cardiac pacemaker, or other objects sensitive to the magnetic field that may pose a danger to both the wearer and the correct operation of magnetic resonance imaging (MRI) equipment; * Concomitant diseases and conditions that, in the Investigator's opinion, can put at risk the patient's safety during his/her participation in the study, or which will influence the safety data analysis in case of the disease/condition exacerbation during the study.
Where this trial is running
Moscow and 4 other locations
- 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 (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, Russia (Not_yet_recruiting)
- V.I. Vernadsky Crimean Federal University — Simferopol, Russia (Recruiting)
- State Budgetary Healthcare Institution Republican Medical Genetic Center — Ufa, Russia (Recruiting)
- State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital — Yekaterinburg, Russia (Recruiting)
Study contacts
- Study coordinator: Svetlana B. Korotkova, MD, PhD
- Email: sbkorotkova@generium.ru
- Phone: +7(495) 988 47 94
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.