Ignite Creation Date:
2024-05-06 @ 3:08 PM
Last Modification Date:
2024-10-26 @ 1:44 PM
Study NCT ID:
NCT04539340
Status:
COMPLETED
Last Update Posted:
2020-10-14
First Post:
2020-08-28
Brief Title:
A Multi-cohort Study of the Tolerance Safety and Pharmacokinetics of GNR-055 in Healthy Volunteers
Sponsor:
AO GENERIUM
Organization:
AO GENERIUM
Study Overview
Official Title:
An Open-label Multi-cohort Dose-escalation Study to Evaluate the Tolerance Safety and Pharmacokinetics of GNR-055 GENERIUM JSC Russia in Healthy Volunteers With a Single Intravenous Administration
Status:
COMPLETED
Status Verified Date:
2020-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 is intended for 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 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 and 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 is a modified enzyme iduronate-2-sulfatase 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
GNR 055 is a modified enzyme iduronate-2-sulfatase 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None