A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Phase 2 — Testing in a larger group (up to a few hundred people) to further evaluate safety and begin to assess effectiveness.
Trial locations(5 sites)
Russia
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
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
V.I. Vernadsky Crimean Federal University, Simferopol
State Budgetary Healthcare Institution Republican Medical Genetic Center, Ufa
State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital, Yekaterinburg