A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients

This study is a retrospective non-interventional study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to evaluate the safety during the treatment and the clinical unmet needs during the treatment in PNH patients in real-world.

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Key information

Conditions

PNH

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Handok

Seoul, South Korea

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patients with PNH aged 18 years or older who started eculizumab treatment in accordance with drug label between 2012 and January 31st, 2020. (However, patients who have received eculizumab as part of a clinical trial or an expanded access program/compassionate use program prior to 2012 are also eligible.)
Patients who are vaccinated against Neisseria meningitidis at least 2 weeks prior to starting eculizumab treatment. Patients who have been treated with appropriate prophylactic antibiotic therapy for 2 weeks after vaccination if they received eculizumab within 2 weeks after the vaccination.

Exclusion criteria

Pediatric patients with PNH aged under 18 years
Patients with hypersensitivity to the active ingredients of eculizumab, murine protein, or other components
Patients with untreated severe meningococcal (Neisseria meningitidis) infection
Patients who received other complement inhibitors for PNH before or during eculizumab treatment

Treatment and study plan

Primary outcomes

Decrease of hemolysis after eculizumab treatment

Time frame: The results of LDH tests performed before eculizumab treatment, at 6, 12, 24 months after the start of eculizumab treatment, and every 12 months thereafter will be collected.

The change in LDH levels before and after eculizumab treatment will be compared to determine whether hemolysis decreased after the treatment.
The Improvement/occurrence of complications and related clinical symptoms before and after eculizumab treatment

Time frame: 6 months before eculizumab administration to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months)

The major complications due to PNH include thromboembolism, pulmonary hypertension, renal failure, and smooth muscle spasm, and their occurrence will be determined by the investigator.

In addition, PNH-related symptoms (fatigue, anemia, dysphagia, shortness of breath/dyspnea, chest pain, abdominal pain, erectile dysfunction, hemoglobinuria, others) before and after eculizumab administration will be compared.
Severity of anemia

Time frame: Before the administration, at 6, 12, 24 months after the start of the treatment and every and 12 months thereafter

Hb levels will be compared to identify the severity of anemia.
The required Unit of pRBC transfusion before and after eculizumab treatment

Time frame: From 1 year before the treatment to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months)
Effectiveness Evaluation at Last Follow-Up

Time frame: At the last follow-up (At the time of data collection retrospectively)

The investigator will perform the final evaluation of the efficacy for entire observation period based on the collected data. The investigator will comprehensively judge and evaluate the decrease of hemolysis occurrence, the improvement and occurrence of complications, and the severity of anemia, etc.

Sponsors and collaborators

Lead sponsor

Handok Inc.

Industry

Registry information

Official study title

A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean Paroxysmal Nocturnal Hemoglobinuria Patients

Important dates

Study start: 2020
Primary completion: 2021
Study completion: 2022
First posted: Apr 20, 2022
Registry last updated: Jan 28, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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