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Clinical trial

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

ID
Name
IDB-MPS-II-III
Description
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.
Trial arms
Trial start
2021-11-30
Estimated PCD
2025-02-01
Trial end
2028-03-01
Status
Recruiting
Phase
Early phase I
Treatment
GNR-055 1.0-2.0-3.0 mg/kg
Weekly IV infusion (lyophilized powder) 1.0-2.0-3.0 mg/kg
Arms:
Adult: GNR-055
Other names:
GNR-055
GNR-055 2.0 mg/kg
Weekly IV infusion (lyophilized powder) 2.0 mg/kg
Arms:
Paediatric: GNR-055 2.0 mg/kg
Other names:
GNR-055
GNR-055 3.0 mg/kg
Weekly IV infusion (lyophilized powder) 3.0 mg/kg
Arms:
Paediatric: GNR-055 3.0 mg/kg
Other names:
GNR-055
Size
32
Primary endpoint
Incidence of Adverse events (AEs) and Serious Adverse Events (SAEs)
Baseline to Week 56
Urine GAG excretion
Baseline to Week 4, 8, 10, 26, and 52
Eligibility criteria
Inclusion Criteria: * 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 Criteria: * 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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Sequential Assignment', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 32, 'type': 'ESTIMATED'}}
Updated at
2024-03-06

1 organization

1 product

2 indications

Organization
AO Generium
Product
GNR-055
Indication
Mucopolysaccharidosis Type II
Indication
Metabolic Diseases