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
2026年8月28日 更新者：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.
調査の概要
状態
募集
研究の種類
観察的
入学 (推定)
20
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究連絡先
- 名前：Ipsen Clinical Study Enquiries
- 電話番号：See e-mail
- メール：clinical.trials@ipsen.com
研究場所
-
-
-
Beijing、中国
- 積極的、募集していない
- Capital Children's Medical Center
-
Chongqing、中国
- 積極的、募集していない
- Chongqing Medical University - Children's Hospital
-
Hunan、中国
- 募集
- Hunan Children's Hospital
-
Nanjing、中国
- 積極的、募集していない
- Nanjing Medical University-Nanjing Children's Hospital
-
Shanghai、中国
- 募集
- Children's Hospital of Fudan University Endocrinology and Metabolism
-
Xiamen、中国
- 積極的、募集していない
- Xiamen Children's Hospital
-
-
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
いいえ
サンプリング方法
非確率サンプル
調査対象母集団
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.
説明
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
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Percentage of participants experiencing adverse events (AEs)
時間枠：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.
|
An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
|
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.
|
|
Percentage of participants experiencing serious adverse events (SAEs)
時間枠：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.
|
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
|
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.
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Event-free survival (EFS)
時間枠：From first ICF signature and up to end of data collection (approximately 5 years of data collection
|
EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection
|
|
Surgical biliary diversion-free survival
時間枠：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Liver transplant-free survival
時間枠：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Overall survival
時間枠：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
Defined as time from the start of odevixibat treatment to death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Pruritus improvement
時間枠：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
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.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Change from baseline in serum bile acid
時間枠：From baseline and up to end of data collection (approximately 5 years of data collection
|
Change from baseline assessed by measuring serum bile acid levels at each patient visit.
|
From baseline and up to end of data collection (approximately 5 years of data collection
|
協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
スポンサー
捜査官
- スタディディレクター：Ipsen Medical Director、Ipsen
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (実際)
2026年4月16日
一次修了 (推定)
2031年4月30日
研究の完了 (推定)
2031年4月30日
試験登録日
最初に提出
2026年5月8日
QC基準を満たした最初の提出物
2026年5月14日
最初の投稿 (実際)
2026年5月15日
学習記録の更新
投稿された最後の更新 (実際)
2026年8月31日
QC基準を満たした最後の更新が送信されました
2026年8月28日
最終確認日
2026年8月1日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- CLIN-60240-032
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
はい
IPD プランの説明
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 共有時間枠
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 共有アクセス基準
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
いいえ
米国FDA規制機器製品の研究
いいえ
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