A Retrospective Chart Review Study to Evaluate the Impact of Eculizumab in Korean PNH Patients

PNH Clinical Trial

Official title:

A Retrospective Chart Review Study to Evaluate the Impact of Eculizumab in Korean Paroxysmal Nocturnal Hemoglobinuria Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05337683
• Other study ID #: HD_ECU_OS2020
• Status: Completed
• Start date: December 11, 2020
• Est. completion date: January 3, 2022

Study information

• Verified date: April 2022
• Source: Handok Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study is a retrospective chart review 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 80
• Est. completion date: January 3, 2022
• Est. primary completion date: August 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• PNH

Locations

Country	Name	City	State
Korea, Republic of	Handok	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Handok Inc.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.
Primary	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)
Primary	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
Primary	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)
Primary	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)