Skip to main content
OpenTrials
Completed

NCT Number: NCT04749810

Observational Study of Elizaria® in aHUS Patients

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Atypical Hemolytic Uremic Syndrome Anemia Anemia, Hemolytic Azotemia Blood Platelet Disorders Cytopenia Disease Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Hematologic Diseases Hemic and Lymphatic Diseases Hemolysis Hemolytic-Uremic Syndrome Kidney Diseases Male Urogenital Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Syndrome Thrombocytopenia Thrombotic Microangiopathies Uremia Urogenital Diseases Urologic Diseases aHUS

Age range

2 month and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation, Kazan', Russia

Loading trial locations.

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Elizaria®

Drug

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

Primary outcomes

  1. Change in platelet count compared to the screening level

    Time frame: 52 week

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

Secondary outcomes

  1. Change in lactate dehydrogenase (LDH) levels from baseline at screening

    Time frame: 52 week

    Change in LDH levels at 52 week after starting study drug treatment from baseline at screening

  2. Proportion of patients with normalized platelet levels

    Time frame: 52 week

    Proportion of patients with normal platelet count at 52 week after initiation of study drug treatment

  3. Proportion of patients with no thrombotic microangiopathy (TMA) events

    Time frame: 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.

  4. Proportion of TMA-related interventions

    Time frame: 52 week

    The proportion of TMA-related interventions is defined as (number of plasma therapy sessions + number of hemodialysis sessions) / number of patient days.

  5. Proportion of patients with complete TMA response

    Time frame: 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

  6. Change in eGFR (ml / min. / 1.73m2) compared with the baseline level at screening;

    Time frame: 52 week

    Change in eGFR (mL/min/1.73m2) at 52 week after initiation of study drug treatment from baseline at screening

  7. 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.

    Time frame: 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

  8. Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to baseline at screening.

    Time frame: 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

  9. Proportion of patients with an increase in hemoglobin level of more than 20 g / l compared to the baseline level at screening.

    Time frame: 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

  10. Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2

    Time frame: 52 week

    Changes in MAC levels at 52 week compared to baseline

  11. The frequency and severity of adverse events (AEs)

    Time frame: 52 weeks

    Frequency and severity of adverse events (AEs), including serious adverse events (SAEs) and AEs associated with study drug use

  12. Proportion of patients with antidrug antibodies

    Time frame: 52 weeks

    Proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity

Sponsors and collaborators

Lead sponsor

AO GENERIUM

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2022
Study completion
2022
First posted
Feb 11, 2021
Registry last updated
Aug 4, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.