NCT06475404

A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Open-label Multi-cohort Study of the Tolerability, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers After a Single Intravenous Injection in Increasing Doses

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: AO GENERIUM · Industry
• Sex: Male
• Age: 18 Years – 45 Years
• Healthy volunteers: Accepted

Summary

It is a phase I open-label single-dose, dose-escalation cohort study to evaluate of the tolerance, safety, and pharmacokinetics of GNR-055 in healthy volunteers

Detailed description

GNR-055 (verenafusp alfa) is intended for enzyme replacement therapy (ERT) in patient with Mucopolysaccharidosis type II (MPS II), or Hunter syndrome. MPS II is a lysosomal storage disease with an X-linked recessive inheritance type, which is characterized by a decrease in the activity of the lysosomal enzyme iduronate-2-sulfatase (I2S), caused by a mutation in the idursulfase (IDS) gene. Enzyme deficiency leads to the accumulation of glycosaminoglycans (GAG) in lysosomes, mainly fractions of heparan and dermatan sulfates. Because of the insufficient activity of iduronate sulfatase participating in the first stage of catabolism of GAG, they accumulate in lysosomes of almost all types of cells of various tissues and organs. The disease is manifested by growth retardation, damage of many organs and systems, severe deformations of bones and joints, gross facial features, pathology of the respiratory and cardiovascular systems, damage to parenchymal organs (hepatosplenomegaly), hearing impairment. A severe form of the disease occurs with the involvement of the nervous system in the pathological process, including mental retardation, behavior anomalies, and impaired motor function. GNR 055 (verenafusp alfa) is a modified enzyme I2S capable of penetrating the blood-brain barrier and thus it is expected to prevent neurodegenerative consequences and the development of cognitive deficit in the future that will allow achieving a significant improvement in the life quality and expectancy of patients with MPS II.

Conditions

• Mucopolysaccharidosis Type II
• Metabolic Diseases

Interventions

Type	Name	Description
BIOLOGICAL	verenafusp alfa 6 mg/kg	Single intravenous administration of 6 mg/kg of verenafusp alfa
BIOLOGICAL	verenafusp alfa 9 mg/kg	Single intravenous administration of 9 mg/kg of verenafusp alfa
BIOLOGICAL	verenafusp alfa 12 mg/kg	Single intravenous administration of 12 mg/kg of verenafusp alfa

Timeline

• Start date: 2023-04-06
• Primary completion: 2023-09-20
• Completion: 2023-09-20
• First posted: 2024-06-26
• Last updated: 2024-07-03

Locations

1 site across 1 country: Russia