Observational Study of Elizaria® in aHUS Patients

NCT04749810 | Completed

• 1 other identifier: ECU-aHUS-N01
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 8

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

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (12)

• Change in lactate dehydrogenase (LDH) levels from baseline at screening

  52 week

• Proportion of patients with normalized platelet levels

  52 week

• Proportion of patients with no thrombotic microangiopathy (TMA) events

  52 week

• Proportion of TMA-related interventions

  52 week

• Proportion of patients with complete TMA response

  52 week

Study Arms (1)

Elizaria®

Eculizumab

Drug: Elizaria®

Interventions

Elizaria® DRUG

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.

Also known as: Eculizumab

Elizaria®

Eligibility Criteria

• Age: 2 Months+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Written informed consent to study participation.
• Male and female patients aged 2 months and older with documented atypical hemolytic uremic syndrome (aHUS)diagnosis.

You may not qualify if:

• \. Intolerance to eculizumab, or other components of the drug.

Sponsors & Collaborators

• AO GENERIUM: lead

Study Sites (8)

Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation

Kazan', 420012, Russia

Location

Regional State Budgetary Healthcare Institution "Krasnoyarsk' Regional Clinical Center for Maternity and Childhood Protection"

Krasnoyarsk, 660074, Russia

Location

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, 107014, Russia

Location

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, 119991, Russia

Location

State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department"

Moscow, 123182, Russia

Location

State budgetary healthcare institution of the city of Moscow "City Clinical Hospital n.a. A.K. Eramishantsev of the Moscow City Healthcare Department"

Moscow, 129327, Russia

Location

St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital"

Saint Petersburg, 191104, Russia

Location

St. Petersburg's State Budgetary Healthcare Institution "Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies"

Saint Petersburg, 198205, Russia

Location

MeSH Terms

Conditions

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

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Hemic and Lymphatic Diseases; Cytopenia; Pathological Conditions, Signs and Symptoms

Study Officials

• Oksana A. Markova, MD

  AO GENERIUM

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 8, 2021
• First Posted: February 11, 2021
• Study Start: December 19, 2019
• Primary Completion: February 28, 2022
• Study Completion: April 30, 2022
• Last Updated: August 4, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

RU (8)