Observational Study of Elizaria® in aHUS Patients

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04749810
• Other study ID #: ECU-aHUS-N01
• Status: Completed
• 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
• 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: April 30, 2022
• Est. primary completion date: February 28, 2022
• 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.

Related Conditions & MeSH terms

• aHUS
• Atypical Hemolytic Uremic Syndrome
• Azotemia
• Hemolysis
• Hemolytic-Uremic Syndrome
• Syndrome

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.

Locations

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

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