A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
2026년 8월 28일 업데이트: Ipsen
Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).
This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.
PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus).
Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC.
The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.
연구 개요
상태
모병
연구 유형
관찰
등록 (추정된)
20
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 연락처
- 이름: Ipsen Clinical Study Enquiries
- 전화번호: See e-mail
- 이메일: clinical.trials@ipsen.com
연구 장소
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Beijing, 중국
- 모집하지 않고 적극적으로
- Capital Children's Medical Center
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Chongqing, 중국
- 모집하지 않고 적극적으로
- Chongqing Medical University - Children's Hospital
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Hunan, 중국
- 모병
- Hunan Children's Hospital
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Nanjing, 중국
- 모집하지 않고 적극적으로
- Nanjing Medical University-Nanjing Children's Hospital
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Shanghai, 중국
- 모병
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Xiamen, 중국
- 모집하지 않고 적극적으로
- Xiamen Children's Hospital
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
아니
샘플링 방법
비확률 샘플
연구 인구
Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) (all types) who have been prescribed odevixibat by their treating physician will be eligible.
Participants who started odevixibat treatment before the implementation of the registry may also be enrolled.
설명
Inclusion Criteria:
- Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician
- On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions)
- Signed informed consent and assent, as appropriate
Exclusion Criteria:
- Currently participating in a clinical trial with odevixibat
- Currently participating in any interventional clinical trial for PFIC
- Have any contraindication to odevixibat as per the approved label in China
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Percentage of participants experiencing adverse events (AEs)
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
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An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
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From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
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Percentage of participants experiencing serious adverse events (SAEs)
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
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SAEs are collected as part of safety reporting and include events meeting seriousness criteria (e.g., death, life-threatening, hospitalization, etc.) as defined in the protocol
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From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Event-free survival (EFS)
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection
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EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection
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Surgical biliary diversion-free survival
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Liver transplant-free survival
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Overall survival
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Pruritus improvement
기간: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Pruritus improvement described at each patient visit using a (semi-)objective scoring scale to assess level of pruritus from the start of the odevixibat treatment.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Change from baseline in serum bile acid
기간: From baseline and up to end of data collection (approximately 5 years of data collection
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Change from baseline assessed by measuring serum bile acid levels at each patient visit.
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From baseline and up to end of data collection (approximately 5 years of data collection
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
스폰서
수사관
- 연구 책임자: Ipsen Medical Director, Ipsen
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작 (실제)
2026년 4월 16일
기본 완료 (추정된)
2031년 4월 30일
연구 완료 (추정된)
2031년 4월 30일
연구 등록 날짜
최초 제출
2026년 5월 8일
QC 기준을 충족하는 최초 제출
2026년 5월 14일
처음 게시됨 (실제)
2026년 5월 15일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2026년 8월 31일
QC 기준을 충족하는 마지막 업데이트 제출
2026년 8월 28일
마지막으로 확인됨
2026년 8월 1일
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- CLIN-60240-032
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
예
IPD 계획 설명
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
IPD 공유 기간
Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU
IPD 공유 액세스 기준
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
아니
미국 FDA 규제 기기 제품 연구
아니
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