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
Who is this study for? Patients with mucopolysaccharidosis II
What treatments are being studied? GNR-055
Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.
Eligibility
Participation Requirements
Sex: Male
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
Russian Federation
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)
NOT_YET_RECRUITING
Moscow
Federal State Budgetary Educational Institution of Higher Education St. Petersburg State Pediatric Medical University of the Ministry of Health of the Russian Federation
NOT_YET_RECRUITING
Saint Petersburg
V.I. Vernadsky Crimean Federal University
RECRUITING
Simferopol
State Budgetary Healthcare Institution Republican Medical Genetic Center
RECRUITING
Ufa
State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital
RECRUITING
Yekaterinburg
Contact Information
Primary
Svetlana B. Korotkova, MD, PhD
sbkorotkova@generium.ru
+7(495) 988 47 94
Backup
Oksana A. Markova, MD, MSc
oamarkova@generium.ru
+7(495) 988 47 94
Time Frame
Start Date: 2021-11-30
Estimated Completion Date: 2028-03
Participants
Target number of participants: 32
Treatments
Experimental: Adult: GNR-055
GNR-055: 1.0-2.0-3.0 mg/kg
Experimental: Paediatric: GNR-055 2.0 mg/kg
GNR-055 2.0 mg/kg
Experimental: Paediatric: GNR-055 3.0 mg/kg
GNR-055 3.0 mg/kg
Related Therapeutic Areas
Sponsors
Leads: AO GENERIUM