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

NCT05337683 | Completed

• 1 other identifier: HD_ECU_OS2020
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

PNH

Outcome Measures

Primary Outcomes (5)

• 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)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Handok Inc.: lead

Study Sites (1)

Handok

Seoul, South Korea

Location

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 8, 2022
• First Posted: April 20, 2022
• Study Start: December 11, 2020
• Primary Completion: August 30, 2021
• Study Completion: January 3, 2022
• Last Updated: January 28, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)