Observational Study of Elizaria® in aHUS Patients

Sponsor

AO GENERIUM (Industry)

Overall Status

Completed

CT.gov ID

NCT04749810

Collaborator

(none)

Enrollment

Locations

28.4

Actual Duration (Months)

6.3

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

It is a multicenter observational non-comparative study of the efficacy and safety of long-term pathogenetic Elizaria® therapy in patients with atypical Hemolytic Uremic Syndrome

Condition or Disease	Intervention/Treatment	Phase
Atypical Hemolytic Uremic Syndrome aHUS	Drug: Elizaria®

Detailed Description

After screening, patients meeting all of the inclusion / non-inclusion criteria and vaccinated against meningococcal infections were treated by Elizaria®.

The study is planned to include at least 50 patients receiving Elizaria® for the aHUS treatment.

The study will consist of a screening period of up to 4 weeks, including, if necessary, immunization with meningococcal vaccine, a treatment period of 52 weeks.

Medication will be prescribed in accordance with routine medical practice. Accordingly to minimize the risks and subjectivity of assessments the methods adopted in the routine practice of treating patients with aHUS will be used.

Investigators enroll patients with aHUS diagnosis who have indications for pathogenetic therapy and who are receiving Elizaria® under the government program. Patients will receive medication in accordance with the established requirements of national standards and protocols for the treatment of patients with aHUS. The registration of the amount of the drug used will be carried out on the basis of information in the Patient Diaries, as well as primary documentation.

Study Design

Study Type:

Observational

Actual Enrollment :

50 participants

Observational Model:

Other

Time Perspective:

Prospective

Official Title:

Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Atypical Hemolytic Uremic Syndrome

Actual Study Start Date :

Dec 19, 2019

Actual Primary Completion Date :

Feb 28, 2022

Actual Study Completion Date :

Apr 30, 2022

Arms and Interventions

Arm	Intervention/Treatment
Elizaria® Eculizumab	Drug: Elizaria® Induction cycle: 900 mg (3 vials of 30 mL, 10 mg/mL) intravenous infusion for 30 minutes once a week for 4 weeks. Maintenance therapy: 1200 mg (4 vials of 30 mL, 10 mg/mL) intravenous infusion for 30 minutes in Week 5, followed by 1200 mg every 14 days. Other Names: Eculizumab

Arm

Intervention/Treatment

Elizaria®

Eculizumab

Drug: Elizaria®

Induction cycle: 900 mg (3 vials of 30 mL, 10 mg/mL) intravenous infusion for 30 minutes once a week for 4 weeks. Maintenance therapy: 1200 mg (4 vials of 30 mL, 10 mg/mL) intravenous infusion for 30 minutes in Week 5, followed by 1200 mg every 14 days.

Other Names:

Eculizumab

Outcome Measures

Primary Outcome Measures

Change in platelet count compared to the screening level [52 week]
Change in platelet count at 52 week after treatment with study drug compared to baseline at screening

Secondary Outcome Measures

Change in lactate dehydrogenase (LDH) levels from baseline at screening [52 week]
Change in LDH levels at 52 week after starting study drug treatment from baseline at screening
Proportion of patients with normalized platelet levels [52 week]
Proportion of patients with normal platelet count at 52 week after initiation of study drug treatment
Proportion of patients with no thrombotic microangiopathy (TMA) events [52 week]
The absence of TMA-related events is defined as the absence, for at least 12 weeks, of: 1) a decrease in platelet counts greater than 25% from baseline at screening; 2) plasma therapy; 3) hemodialysis.
Proportion of TMA-related interventions [52 week]
The proportion of TMA-related interventions is defined as (number of plasma therapy sessions + number of hemodialysis sessions) / number of patient days.
Proportion of patients with complete TMA response [52 week]
Complete TMA response is defined as the absence of abnormalities in LDH and platelet levels + improvement in renal function (decrease in creatinine levels by 25% or more compared to the baseline value on screening) when performed at least two consecutive tests within 8 weeks
Change in eGFR (ml / min. / 1.73m2) compared with the baseline level at screening; [52 week]
Change in eGFR (mL/min/1.73m2) at 52 week after initiation of study drug treatment from baseline at screening
Proportion of patients with an improvement in glomerular filtration rate (eGFR) of 15 ml / min / 1.73m2 or more compared to the baseline level at screening. [52 week]
Proportion of patients with improvement in eGFR of 15 ml/min/1.73m2 or more at 52 week after treatment with study drug compared to baseline at screening
Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to baseline at screening. [52 week]
Proportion of patients with >=1 stage improvement in CKD at 52 week after initiation of study drug treatment compared with baseline at screening
Proportion of patients with an increase in hemoglobin level of more than 20 g / l compared to the baseline level at screening. [52 week]
Proportion of patients with an increase in hemoglobin level of more than 20 g/l at 52 week after the start of study drug treatment compared with baseline at screening
Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2 [52 week]
Changes in MAC levels at 52 week compared to baseline
The frequency and severity of adverse events (AEs) [52 weeks]
Frequency and severity of adverse events (AEs), including serious adverse events (SAEs) and AEs associated with study drug use
Proportion of patients with antidrug antibodies [52 weeks]
Proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Months and Older

Sexes Eligible for Study:

All

Inclusion Criteria

Written informed consent to study participation.
Male and female patients aged 2 months and older with documented atypical hemolytic uremic syndrome (aHUS)diagnosis.
By the time of inclusion in the study, Elizaria® should be prescribed as a pathogenetic therapy for aHUS; Exclusion Criteria
Intolerance to eculizumab, or other components of the drug.

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation	Kazan	Russian Federation	420012
2	Regional State Budgetary Healthcare Institution "Krasnoyarsk' Regional Clinical Center for Maternity and Childhood Protection"	Krasnoyarsk	Russian Federation	660074
3	State budgetary healthcare institution of the city of Moscow "Children's City Clinical Hospital of St. Vladimir of the Healthcare Department of the City of Moscow"	Moscow	Russian Federation	107014
4	Federal State Autonomous Educational Institution of Higher Education "First Moscow State Medical University n.a. I.M. Sechenov" of the Ministry of Health of the Russian Federation	Moscow	Russian Federation	119991
5	State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department"	Moscow	Russian Federation	123182
6	State budgetary healthcare institution of the city of Moscow "City Clinical Hospital n.a. A.K. Eramishantsev of the Moscow City Healthcare Department"	Moscow	Russian Federation	129327
7	St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital"	Saint-Petersburg	Russian Federation	191104
8	St. Petersburg's State Budgetary Healthcare Institution "Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies"	Saint-Petersburg	Russian Federation	198205