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

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

Study Overview

• Status: Completed
• Conditions: PNH

• Study Type: Observational
• Enrollment (Actual): 80

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Handok

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with PNH aged 18 years or older who started receiving eculizumab treatment accordance with the drug label until January 31, 2020 will be enrolled in this study.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Decrease of hemolysis after eculizumab treatment	The change in LDH levels before and after eculizumab treatment will be compared to determine whether hemolysis decreased after the treatment.	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 Improvement/occurrence of complications and related clinical symptoms before and after eculizumab treatment	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.	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)
Severity of anemia	Hb levels will be compared to identify the severity of anemia.	Before the administration, at 6, 12, 24 months after the start of the treatment and every and 12 months thereafter
The required Unit of pRBC transfusion before and after eculizumab treatment		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	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.	At the last follow-up (At the time of data collection retrospectively)

Collaborators and Investigators

• Sponsor: Handok Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 11, 2020
• Primary Completion (Actual): August 30, 2021
• Study Completion (Actual): January 3, 2022

Study Registration Dates

• First Submitted: April 8, 2022
• First Submitted That Met QC Criteria: April 14, 2022
• First Posted (Actual): April 20, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 24, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Other Study ID Numbers: HD_ECU_OS2020

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No