Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®

NCT04671810 | Completed

• 1 other identifier: ECU-PNH-N01
• Study Type: observational
• Target: 44
• Locations: 1 country
• Sites: 8

Brief Summary

This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Dynamics of the peripheral blood LDH level during 52 weeks of the maintenance stage of treatment with Elizaria® (between Visits 6 and 13, inclusive).

  To assess the dynamics of the peripheral blood LDH level during 52 weeks the area under the LDH level - time curve (AUC) will be calculated. Descriptive statistics will be provided for the values of the LDH level at each visit, as well as for the AUC, indicating the geometric means and coefficient of variation.

  52-56 weeks

Secondary Outcomes (9)

• Changes in hemoglobin levels during maintenance therapy with Elizaria® (between Visits 6 and 13 inclusive)

  52-56 weeks

• Number / proportion of patients with a change in the level of hemoglobin in the blood ± 5 g / l and ± 10 g / l. during the period of maintenance therapy with Elizaria® (between Visits 6 and 13 inclusive)

  52-56 weeks

• The number / proportion of patients with various thrombotic complications that developed during treatment with Elizaria®.

  52-56 weeks

• The number / proportion of patients requiring transfusion of erythrocyte components of donated blood during treatment with Elizaria®.

  52-56 weeks

• Number/proportion of patients with breakthrough hemolysis while observing an increased LDH, after a previous decrease in the course of treatment.

  52-56 weeks

Interventions

Elizaria® BIOLOGICAL

Elizaria® will be prescribed by the Physician according to the actual SMPC.

Also known as: Eculizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The population of the study is the patients with an established diagnosis of PNH who have indications for pathogenic treatment and are receiving Elizaria®.

You may qualify if:

• Male and female patients aged 2 years with an established diagnosis of PNH;
• Prescribing Elizaria® as a pathogenetic therapy;
• Signing by the patient of the informed consent form for participation in the study.

You may not qualify if:

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

Sponsors & Collaborators

• AO GENERIUM: lead

Study Sites (8)

State budgetary healthcare institution "Chelyabinsk Regional Clinical Hospital"

Chelyabinsk, Chelyabinsk Oblast, 454076, Russia

Location

State Budgetary Institution of Healthcare Irkutsk Order of the "Badge of Honor" Regional Clinical Hospital

Irkutsk, Irkutsk Oblast, 644079, Russia

Location

State Budgetary Institution of Healthcare of the City of Moscow City Clinical Hospital named after S.P. Botkin of the Moscow Department of Health

Moscow, Moscow Oblast, 125284, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "Novosibirsk State Medical University" of the Ministry of Health of the Russian Federation on the basis of the State Budgetary Institution of Health of the Novosibirsk Region "City Clinic

Novosibirsk, Novosibirsk Oblast, 630091, Russia

Location

State budgetary institution of health care of the Republic of Karelia "Republican hospital named after V.A. Baranov "

Petrozavodsk, Republic of Karelia, 185019, Russia

Location

State budgetary institution of the Rostov region "Rostov Regional Clinical Hospital"

Rostov-on-Don, Rostov Oblast, 344015, Russia

Location

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

Samara, Samara Oblast, 443099, Russia

Location

State Budgetary Healthcare Institution of the Sverdlovsk Region "Sverdlovsk Regional Clinical Hospital No. 1"

Yekaterinburg, Sverdlovsk Oblast, 620102, Russia

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Oksana Markova, MD

  AO GENERIUM

  STUDY CHAIR

Study Design

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

Study Record Dates

• First Submitted: December 1, 2020
• First Posted: December 17, 2020
• Study Start: June 13, 2019
• Primary Completion: December 28, 2022
• Study Completion: May 5, 2023
• Last Updated: September 13, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

RU (8)