Phase 2 open-label efficacy and safety study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with progressive familial intrahepatic cholestasis: 48-week interim efficacy analysis

被引：0

作者：

Thompson, Richard J. ^{[1
]}

Kelly, Deirdre A. ^{[2
]}

McClean, Patricia ^{[3
]}

Miethke, Alexander G. ^{[4
,5
]}

Soufi, Nisreen ^{[6
,7
]}

Rivet, Christine ^{[8
]}

Jankowska, Irena ^{[9
]}

Mack, Cara L. ^{[10
]}

Karthikeyan, Palaniswamy ^{[1
]}

Gu, Joan ^{[11
]}

Jaecklin, Thomas ^{[12
]}

Squires, Robert H. ^{[13
,14
]}

Loomes, Kathleen M. ^{[15
,16
]}

机构：

[1] Kings Coll London, Inst Liver Studies, London, England

[2] Birmingham Childrens Hosp, Liver Unit, Birmingham, W Midlands, England

[3] Leeds Gen Infirm, Childrens Liver Unit, Leeds, W Yorkshire, England

[4] Cincinnati Childrens Hosp Med Ctr, Div Gastroenterol Hepatol & Nutr, Cincinnati, OH 45229 USA

[5] Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA

[6] Childrens Hosp Los Angeles, Div Gastroenterol Hepatol & Nutr, Los Angeles, CA 90027 USA

[7] Univ Southern Calif, Keck Sch Med, Dept Pediat, Los Angeles, CA USA

[8] Hop Femme Mere Enfant, Serv Hepatol Gastroenterol & Nutr Pediat, Bron, France

[9] Childrens Mem Hlth Inst, Dept Gastroenterol Hepatol Feeding Disorders & Pe, Warsaw, Poland

[10] Univ Colorado, Childrens Hosp Colorado, Sch Med, Sect Pediat Gastroenterol Hepatol & Nutr, Aurora, CO USA

[11] Shire, Lexington, MA USA

[12] Shire, Zug, Switzerland

[13] Univ Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Pediat Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA

[14] Univ Pittsburgh, Sch Med, Dept Pediat, Pittsburgh, PA 15261 USA

[15] Childrens Hosp Philadelphia, Div Gastroenterol Hepatol & Nutr, Philadelphia, PA 19104 USA

[16] Univ Penn, Dept Pediat, Perelman Sch Med, Philadelphia, PA 19104 USA

来源：

HEPATOLOGY | 2017年 / 66卷

关键词：

D O I：

暂无

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

引用

页码：57A / 57A

页数：1

共 26 条

[1] Phase 2 open-label study with a placebo-controlled drug withdrawal period of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille Syndrome: 48-week interim efficacy analysis
Gonzales, Emmanuel
Sturm, Ekkehard
Stormon, Michael
Sokal, Etienne
Hardikar, Winita
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Jaecklin, Thomas
Gu, Joan
Desai, Nirav K.
Dorenbaum, Alejandro
Kennedy, Ciara
Baker, Alastair
Jacquemin, Emmanuel
JOURNAL OF HEPATOLOGY, 2019, 70 (01) : E119 - E120
[2] Growth analysis in children with progressive familial intrahepatic cholestasis treated with the apical sodium-dependent bile acid transporter inhibitor maralixibat
Thompson, Richard
Kelly, Deirdre
Rajwal, Sanjay
Miethke, Alexander
Soufi, Nisreen
Rivet, Christine
Jankowska, Irena
Mack, Cara
Jaecklin, Thomas
Squires, Robert
Loomes, Kathleen
JOURNAL OF HEPATOLOGY, 2019, 70 (01) : E131 - E132
[3] A long-term phase 2 safety and efficacy study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille syndrome: preliminary results from the IMAGINE study
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Gu, Joan
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McClean, Patricia
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Thompson, Richard J.
HEPATOLOGY, 2017, 66 (06) : 1255A - 1256A
[4] Safety, tolerability and efficacy of volixibat, an apical sodium-dependent bile acid transporter inhibitor, in adults with non-alcoholic steatohepatitis: 24-week interim analysis results from a phase 2 study
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[6] Multicenter, randomized, open-label study of the safety and efficacy of histamine dihydrochloride and interferon alpha-2b for the treatment of chronic hepatitis C: 48-week results
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[7] Loss of FIC1 in progressive familial intrahepatic cholestasis type 1 (PFIC1) is associated with decreased farnesoid X receptor (FXR) expression and ileal overexpression of the apical sodium-dependent bile acid transporter (ASBT).
Shneider, B
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[8] Safety, efficacy, and pharmacokinetics of bedaquiline in Japanese patients with pulmonary multidrug-resistant tuberculosis: An interim analysis of an open-label, phase 2 study
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[9] IMPROVED QUALITY OF LIFE IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS FOLLOWING 24 WEEKS OF TREATMENT WITH ODEVIXIBAT, AN ILEAL BILE ACID TRANSPORTER INHIBITOR: RESULTS FROM THE PHASE 3 PEDFIC 1 STUDY
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VALUE IN HEALTH, 2021, 24 : S155 - S155
[10] Safety and efficacy of denosumab for adults and skeletally mature adolescents with giant cell tumour of bone: interim analysis of an open-label, parallel-group, phase 2 study
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