Clinical trial NCT04749810: Observational Study of Elizaria® in aHUS Patients
Official title: Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Atypical Hemolytic Uremic Syndrome
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Atypical Hemolytic Uremic Syndrome
aHUS
Keywords: Atypical Hemolytic Uremic Syndrome
Azotemia
Hemolytic-Uremic Syndrome
Hemolysis
Syndrome
Disease
Pathologic Processes
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Lead sponsor: AO GENERIUM
Sponsor class: INDUSTRY
First posted: 2021-02-11
Enrollment: 50
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: At least 50 adult and pediatric patients (from 2 months), with a confirmed diagnosis of aHUS, who meet the enrollment criteria.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in platelet count compared to the screening level
timeFrame: 52 week
Secondary outcomes:
measure: Change in lactate dehydrogenase (LDH) levels from baseline at screening
timeFrame: 52 week
measure: Proportion of patients with normalized platelet levels
timeFrame: 52 week
measure: Proportion of patients with no thrombotic microangiopathy (TMA) events
timeFrame: 52 week
measure: Proportion of TMA-related interventions
timeFrame: 52 week
measure: Proportion of patients with complete TMA response
timeFrame: 52 week
measure: Change in eGFR (ml / min. / 1.73m2) compared with the baseline level at screening;
timeFrame: 52 week
measure: 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.
timeFrame: 52 week
measure: Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to baseline at screening.
timeFrame: 52 week
measure: Proportion of patients with an increase in hemoglobin level of more than 20 g / l compared to the baseline level at screening.
timeFrame: 52 week
measure: Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2
timeFrame: 52 week
measure: The frequency and severity of adverse events (AEs)
timeFrame: 52 weeks
measure: Proportion of patients with antidrug antibodies
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Kazan
country: Russian Federation
facility: Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation
city: Krasnoyarsk
country: Russian Federation
facility: Regional State Budgetary Healthcare Institution "Krasnoyarsk' Regional Clinical Center for Maternity and Childhood Protection"
city: Moscow
country: Russian Federation
facility: 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"
city: Moscow
country: Russian Federation
facility: 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
city: Moscow
country: Russian Federation
facility: State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department"
city: Moscow
country: Russian Federation
facility: State budgetary healthcare institution of the city of Moscow "City Clinical Hospital n.a. A.K. Eramishantsev of the Moscow City Healthcare Department"
city: Saint-Petersburg
country: Russian Federation
facility: St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital"
city: Saint-Petersburg
country: Russian Federation
facility: St. Petersburg's State Budgetary Healthcare Institution "Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies"
Study officials:
affiliation: AO GENERIUM
name: Oksana A. Markova, MD
role: STUDY_CHAIR
References: