Atypical Hemolytic Uremic Syndrome Clinical Trial
Official title:
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Atypical Hemolytic Uremic Syndrome
Verified date | February 2022 |
Source | AO GENERIUM |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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
Status | Completed |
Enrollment | 50 |
Est. completion date | April 30, 2022 |
Est. primary completion date | February 28, 2022 |
Accepts healthy volunteers | |
Gender | All |
Age group | 2 Months and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
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 | 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 | 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 | State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department" | Moscow | |
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 | St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital" | Saint-Petersburg |
Lead Sponsor | Collaborator |
---|---|
AO GENERIUM |
Russian Federation,
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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