Observational Study of Elizaria® in aHUS Patients

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

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

Study Overview

• Status: Completed
• Conditions: Atypical Hemolytic Uremic Syndrome; aHUS
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

Study Locations

• Russian Federation
  • Kazan, Russian Federation, 420012
    • Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation
  • Krasnoyarsk, Russian Federation, 660074
    • Regional State Budgetary Healthcare Institution "Krasnoyarsk' Regional Clinical Center for Maternity and Childhood Protection"
  • Moscow, Russian Federation, 107014
    • 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, 119991
    • 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, 123182
    • State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department"
  • Moscow, Russian Federation, 129327
    • State budgetary healthcare institution of the city of Moscow "City Clinical Hospital n.a. A.K. Eramishantsev of the Moscow City Healthcare Department"
  • Saint-Petersburg, Russian Federation, 191104
    • St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital"
  • Saint-Petersburg, Russian Federation, 198205
    • St. Petersburg's State Budgetary Healthcare Institution "Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies"

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

At least 50 adult and pediatric patients (from 2 months), with a confirmed diagnosis of aHUS, who meet the enrollment criteria.

Description

Inclusion Criteria

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

1. Intolerance to eculizumab, or other components of the drug.

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in platelet count compared to the screening level	Change in platelet count at 52 week after treatment with study drug compared to baseline at screening	52 week

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in lactate dehydrogenase (LDH) levels from baseline at screening	Change in LDH levels at 52 week after starting study drug treatment from baseline at screening	52 week
Proportion of patients with normalized platelet levels	Proportion of patients with normal platelet count at 52 week after initiation of study drug treatment	52 week
Proportion of patients with no thrombotic microangiopathy (TMA) events	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.	52 week
Proportion of TMA-related interventions	The proportion of TMA-related interventions is defined as (number of plasma therapy sessions + number of hemodialysis sessions) / number of patient days.	52 week
Proportion of patients with complete TMA response	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	52 week
Change in eGFR (ml / min. / 1.73m2) compared with the baseline level at screening;	Change in eGFR (mL/min/1.73m2) at 52 week after initiation of study drug treatment from baseline at screening	52 week
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.	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	52 week
Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to baseline at screening.	Proportion of patients with >=1 stage improvement in CKD at 52 week after initiation of study drug treatment compared with baseline at screening	52 week
Proportion of patients with an increase in hemoglobin level of more than 20 g / l compared to the baseline level at screening.	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	52 week
Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2	Changes in MAC levels at 52 week compared to baseline	52 week
The frequency and severity of adverse events (AEs)	Frequency and severity of adverse events (AEs), including serious adverse events (SAEs) and AEs associated with study drug use	52 weeks
Proportion of patients with antidrug antibodies	Proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity	52 weeks

Collaborators and Investigators

• Sponsor: AO GENERIUM

Study record dates

Study Major Dates

• Study Start (Actual): December 19, 2019
• Primary Completion (Actual): February 28, 2022
• Study Completion (Actual): April 30, 2022

Study Registration Dates

• First Submitted: February 8, 2021
• First Submitted That Met QC Criteria: February 8, 2021
• First Posted (Actual): February 11, 2021

Study Record Updates

• Last Update Posted (Actual): August 4, 2022
• Last Update Submitted That Met QC Criteria: August 2, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Keywords: Pathologic Processes; Anemia; Hematologic Diseases; Urologic Diseases; Kidney Diseases; Syndrome; Blood Platelet Disorders; Disease; Hemolysis; Atypical Hemolytic Uremic Syndrome; Thrombocytopenia; Hemolytic-Uremic Syndrome; Thrombotic Microangiopathies; Azotemia; Anemia, Hemolytic; Uremia
• Additional Relevant MeSH Terms: Pathologic Processes; Kidney Diseases; Urologic Diseases; Disease; Hematologic Diseases; Anemia; Thrombocytopenia; Blood Platelet Disorders; Anemia, Hemolytic; Thrombotic Microangiopathies; Uremia; Syndrome; Azotemia; Hemolysis; Hemolytic-Uremic Syndrome; Atypical Hemolytic Uremic Syndrome; Physiological Effects of Drugs; Immunosuppressive Agents; Immunologic Factors; Complement Inactivating Agents; Eculizumab
• Other Study ID Numbers: ECU-aHUS-N01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No