Status:
RECRUITING
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Lead Sponsor:
AO GENERIUM
Conditions:
Mucopolysaccharidosis Type II
Metabolic Diseases
Eligibility:
MALE
Phase:
PHASE2
PHASE3
Brief 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.
Detailed Description
GNR-055 is intended for ERT in patient with Mucopolysaccharidosis type II (MPS II), or Hunter syndrome. MPS II is a recessive X-linked inheritance lysosomal storage disease, which is characterized by ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 30 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2028
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT05208281
Start Date
November 30 2021
End Date
March 1 2028
Last Update
July 30 2025
Active Locations (5)
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1
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, 119333
2
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, 194100
3
V.I. Vernadsky Crimean Federal University
Simferopol, Russia, 295007
4
State Budgetary Healthcare Institution Republican Medical Genetic Center
Ufa, Russia, 450076