Clinical trial NCT05337683: A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients
Official title: A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean Paroxysmal Nocturnal Hemoglobinuria Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: PNH
Lead sponsor: Handok Inc.
Sponsor class: INDUSTRY
First posted: 2022-04-20
Enrollment: 80
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Decrease of hemolysis after eculizumab treatment
timeFrame: 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.
measure: The Improvement/occurrence of complications and related clinical symptoms before and after eculizumab treatment
timeFrame: 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)
measure: Severity of anemia
timeFrame: Before the administration, at 6, 12, 24 months after the start of the treatment and every and 12 months thereafter
measure: The required Unit of pRBC transfusion before and after eculizumab treatment
timeFrame: 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)
measure: Effectiveness Evaluation at Last Follow-Up
timeFrame: At the last follow-up (At the time of data collection retrospectively)
Secondary outcomes:
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Handok
Study officials:
References: