A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With 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
AO GENERIUM
32 participants
Nov 30, 2021
INTERVENTIONAL
Conditions
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
Inclusion Criteria5
- 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 Criteria4
- 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.
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Interventions
Weekly IV infusion (lyophilized powder) 1.0-2.0-3.0 mg/kg
Weekly IV infusion (lyophilized powder) 2.0 mg/kg
Weekly IV infusion (lyophilized powder) 3.0 mg/kg
Locations(5)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT05208281