A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexMale
AgeNot listed
SponsorAO GENERIUM
About this trial
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 criteria
Qualifiers
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;
Disqualifiers
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.
Trial design
Treatments tested in this trial
- GNR-055 1.0-2.0-3.0 mg/kg
- GNR-055 2.0 mg/kg
- GNR-055 3.0 mg/kg
Treatment groups
32 Participants
are divided into 3 treatment groupsLocations
5Federal 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)119333, Moscow Russia
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation194100, Saint Petersburg Russia
V.I. Vernadsky Crimean Federal University295007, Simferopol Russia
State Budgetary Healthcare Institution Republican Medical Genetic Center450076, Ufa Russia
Sponsors and collaborators
AO GENERIUM
Lead sponsor
Source ClinicalTrials.gov