Ignite Creation Date:
2024-05-06 @ 3:34 PM
Last Modification Date:
2024-10-26 @ 1:52 PM
Study NCT ID:
NCT04671810
Status:
COMPLETED
Last Update Posted:
2023-09-13
First Post:
2020-12-01
Brief Title:
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria
Sponsor:
AO GENERIUM
Organization:
AO GENERIUM
Study Overview
Official Title:
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria in Patients With Paroxysmal Nocturnal Hemoglobinuria
Status:
COMPLETED
Status Verified Date:
2023-09
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:
NAP
Brief Summary:
This is prospective observational study of long-term pathogenic treatment of Elizaria in patients with paroxysmal nocturnal hemoglobinuria
Detailed Description:
Paroxysmal nocturnal hemoglobinuria PNH is an ultra-rare acquired life-threatening progressive clonal blood disease that is developed as a result of the expansion of one or more clones of hematopoietic stem cells with a somatic PIG A gene mutation The main pathogenic mechanism of PNH development is a dysregulation of the complement system in which there are no complement inhibitors CD55 and CD59 that are bounded by the GPI-anchor on the surface of blood cells or there is a deficiency of them
Eculizumab is the first drug for the pathogenetic therapy of PNH a recombinant humanized monoclonal antibody against the C5 component of complement Binding with high affinity to C5 eculizumab prevents the cleavage of C5 into C5a and C5b thereby inhibiting the formation of pro-inflammatory cytokines via C5a and Membrane attack complex MAC via C5b
Using of eculizumab in patients with PNH leads to a significant decrease in hemolysis the incidence of thrombosis and increasing of the quality of life of patients It increases life expectancy with long-term using of eculizumab
The drug Eculizumab developed by JSC GENERIUM Russia is a biological analogue biosimilar of the original drug Soliris
The population of the study is the patients with an established diagnosis of PNH who have indications for pathogenic treatment and are receiving Elizaria
The study duration for each patient will be at least 54 weeks for previously treated patients and 58 weeks for previously untreated patients including the Screening period and the observation period
The study will include the Screening Period and the Observation Period
Screening Period - 2 weeks Days 1 - 14
Follow-up is 52 weeks for previously treated patients and 56 weeks for previously untreated patients
The screening period can be extended until the results of laboratory and instrumental studies are obtained but no more than 14 days
During the observation period for the purpose of routine examination it is planned to conduct a number of intermediate visits approximately every 8 weeks an acceptable interval of 6-10 weeks between the Screening Visit Visit 1 and the Study End Visit Visit 13 in accordance with the plan of the dispensary observation of the patient Additional visits and examinations may be carried out by the decision of the research physician
If the patient is previously untreated the patient undergoes induction therapy with Elizaria at a dose of 600 mg weekly for 4 weeks with a further transition to maintenance therapy at a dose of 900 mg from the 5th week and then every 2 weeks
Eculizumab is the first drug for the pathogenetic therapy of PNH a recombinant humanized monoclonal antibody against the C5 component of complement Binding with high affinity to C5 eculizumab prevents the cleavage of C5 into C5a and C5b thereby inhibiting the formation of pro-inflammatory cytokines via C5a and Membrane attack complex MAC via C5b
Using of eculizumab in patients with PNH leads to a significant decrease in hemolysis the incidence of thrombosis and increasing of the quality of life of patients It increases life expectancy with long-term using of eculizumab
The drug Eculizumab developed by JSC GENERIUM Russia is a biological analogue biosimilar of the original drug Soliris
The population of the study is the patients with an established diagnosis of PNH who have indications for pathogenic treatment and are receiving Elizaria
The study duration for each patient will be at least 54 weeks for previously treated patients and 58 weeks for previously untreated patients including the Screening period and the observation period
The study will include the Screening Period and the Observation Period
Screening Period - 2 weeks Days 1 - 14
Follow-up is 52 weeks for previously treated patients and 56 weeks for previously untreated patients
The screening period can be extended until the results of laboratory and instrumental studies are obtained but no more than 14 days
During the observation period for the purpose of routine examination it is planned to conduct a number of intermediate visits approximately every 8 weeks an acceptable interval of 6-10 weeks between the Screening Visit Visit 1 and the Study End Visit Visit 13 in accordance with the plan of the dispensary observation of the patient Additional visits and examinations may be carried out by the decision of the research physician
If the patient is previously untreated the patient undergoes induction therapy with Elizaria at a dose of 600 mg weekly for 4 weeks with a further transition to maintenance therapy at a dose of 900 mg from the 5th week and then every 2 weeks
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