Observational Study of Elizaria® in aHUS Patients
- Conditions
- aHUSAtypical Hemolytic Uremic Syndrome
- Interventions
- Drug: Elizaria®
- Registration Number
- NCT04749810
- Lead Sponsor
- AO GENERIUM
- 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
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 50
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Elizaria® Elizaria® Eculizumab
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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
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 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 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
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
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 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.
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
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 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
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
Trial Locations
- Locations (8)
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
St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital"
🇷🇺Saint-Petersburg, Russian Federation
State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department"
🇷🇺Moscow, Russian Federation
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
Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation
🇷🇺Kazan, Russian Federation
Regional State Budgetary Healthcare Institution "Krasnoyarsk' Regional Clinical Center for Maternity and Childhood Protection"
🇷🇺Krasnoyarsk, Russian Federation
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
St. Petersburg's State Budgetary Healthcare Institution "Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies"
🇷🇺Saint-Petersburg, Russian Federation