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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04749810
Other study ID # ECU-aHUS-N01
Secondary ID
Status Completed
Phase
First received
Last updated
Start date December 19, 2019
Est. completion date April 30, 2022

Study information

Verified date February 2022
Source AO GENERIUM
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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


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.


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Study Design


Related Conditions & MeSH terms


Intervention

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.

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Sponsors (1)

Lead Sponsor Collaborator
AO GENERIUM

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary Proportion of patients with antidrug antibodies Proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity 52 weeks
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