An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea (ESR-25-22871)
This multicenter, retrospective observational cohort study aims to evaluate the long-term real-world effectiveness and safety of ravulizumab in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in South Korea over a 4-year post-marketing period. By analyzing electronic medical records (EMR) from participating institutions, the study investigates intravascular hemolysis control, transfusion avoidance, breakthrough hemolysis (BTH) characteristics, and overall clinical outcomes in an Asian population.
연구 개요
연구 유형
등록 (추정된)
연락처 및 위치
연구 장소
-
-
-
Seoul, 대한민국
- Yonsei University College of Medicine
-
-
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
Korean patients with Paroxysmal Nocturnal Hemoglobinuria whose retrospective clinical data are available in the electronic medical records (EMR) of the participating hospitals.
설명
Inclusion Criteria:
- Adult patients (18 years of age or older) with a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) documented by flow cytometry.
- Patients who initiated ravulizumab treatment on or before February 28, 2025, and have at least 6 months of follow-up data available.
- To reflect real-world clinical practice, meningococcal (Neisseria meningitidis) vaccination status will be collected if available, but is not a mandatory requirement for study inclusion.
Exclusion Criteria:
- Less than 18 years of age at the time of ravulizumab treatment initiation.
- Known hypersensitivity to ravulizumab or any of its excipients.
- Untreated Neisseria meningitidis infection at the time of ravulizumab treatment initiation.
- Prior or concomitant use of other complement inhibitors for PNH (with the exception of prior eculizumab use).
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: COHORT
- 시간 관점: RETROSPECTIVE
코호트 및 개입
그룹/코호트 |
|---|
|
PNH patients treated with Ravulizumab
Patients diagnosed with PNH and prescribed Ravulizumab at multiple centers in South Korea, whose electronic medical records (EMR) are available for retrospective review. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Proportion of patients achieving lactate dehydrogenase (LDH) levels less than or equal to 1.5 times the upper limit of normal (ULN)
기간: Up to 4 years after ravulizumab initiation
|
Assessment of intravascular hemolysis control, defined as achieving and maintaining serum LDH levels at or below 1.5 times the ULN during the 4-year post-marketing observation period. |
Up to 4 years after ravulizumab initiation
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Incidence and clinical characteristics of breakthrough hemolysis (BTH)
기간: hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
|
BTH is defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin less than 10 g/dL], major adverse vascular events [MAVE, including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) levels of at least 2 times the upper limit of normal (ULN), after prior reduction of LDH to less than 1.5 times the ULN during therapy. Associated contextual factors, including complement-amplifying conditions, hospitalizations, transfusions, and the use of rescue medication, will also be recorded and characterized at each event. |
hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
|
공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- 4-2026-0786
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .