30 closest matches · ranked by relevance
Bile Acid Synthesis Defect · Inborn Error of Bile Acid Metabolism · Inborn Error of Bile Acid Conjugation
Primary: Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation. — 5 Participants
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants
Hepatic Steatosis
Primary: Hepatic Triglyceride (%) — 15.9; 14.8 Fat/Fat+Water (%) — p=0.42
Progressive Familial Intrahepatic Cholestasis · Alagille Syndrome · Cholestatic Liver Disease
Primary: Frequency of Treatment-emergent Adverse Events [TEAEs] — 16; 10; 4; 3 Participants
Alagille Syndrome
Primary: Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level — -82.864; -49.388; -66.126; -42.157 umol/L — p=0.5740
Bile Acid Malabsorption · Cholelithiasis
Primary: Change in Stimulated Fibroblast Growth Factor 19 (FGF19) From Baseline Before Versus Post Cholecystectomy — 81; 186 pg per mL — p=0.03
Infantile Refsum's Disease · Zellweger Syndrome · Adrenoleukodystrophy
Primary: Number of Participants With Excretion of Atypical Bile Acids in Urine by Category — 10; 51; 11; 9 Participants
Primary Sclerosing Cholangitis (PSC)
Primary: DB Phase: Change From Baseline In Serum Alkaline Phosphatase (ALP) — -105.05; -110.19; -26.76 U/L — p=0.0434
Pruritus
Primary: Change From Baseline in the Worst Itch Numeric Rating Scale (WI-NRS) Score up to Week 6 — -2.20; -1.75 scores on a scale — p=0.4577
Cholestatic Liver Disease
Primary: Incidence of Treatment-Emergent Adverse Events — 36; 12; 9; 4 Participants
Bile Acid Synthesis Defect
Primary: Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry) — 25; 6; 10; 10 Participants
Progressive Familial Intrahepatic Cholestasis (PFIC)
Primary: Change From Baseline to Endpoint (Week 13) in Fasting sBA Level — -23; 18; -38 umol/L
Cystic Fibrosis · Liver Fibrosis
Primary: Elastographic Value in kPa Measured by Fibroscan — 4.7 kPa
Primary Sclerosing Cholangitis
Primary: Number of Participants With Treatment Emergent Adverse Events — 21; 10; 16; 15 Participants
Primary Biliary Cholangitis · Cardiovascular Abnormalities
Primary: The Incidence of Cardiac Events — 0; 0; 0; 0 Participants — p=0.001
Nonalcoholic Fatty Liver Disease (NAFLD)
Primary: Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) — 25; 18 participants — p=0.34
Liver Diseases
Primary: Aspartate Transaminase (AST) Change — -6.32 U/L — p=0.03
Biliary Atresia
Primary: Number of Patients With Biliary Atresia (BA) Achieving Total Serum Bile Acids Less Than or Equal to 10 *U*Mol/L Within 24 Weeks of Kasai Portoenterostomy (KP) — 0…
Primary Biliary Cholangitis · Liver Stiffness
Primary: Change in ALP at Week 24 Compared to Baseline — 0.89; 0.84; 1.03 ratio — p=0.0206
Parenteral Nutrition Associated Liver Disease · Short Bowel Syndrome · Gastrointestinal Disease
Primary: Reversal of Cholestasis, Defined as a Direct Bilirubin to <= 2.0 mg/dL. — 11.7; 6.4 Weeks — p==0.001
Cholestasis of Parenteral Nutrition
Primary: Number of Participants With Cholestasis — 1; 3; 7 Participants — p=0.590
Primary Biliary Cirrhosis
Primary: Change in Serum Alkaline Phosphatase Levels. — -57.3 U/L — p=0.001
Biliary Atresia
Primary: The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy — 58.6; 48.6 percentage of participants — p=0.43
Primary Biliary Cirrhosis
Primary: Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration — -0.38 mmol/L
Familial Partial Lipodystrophy
Primary: Change in the Liver Triglycerides (TG). — 13.2; 6.4; 12.3; 10.6 percentage of liver triglycerides
Parenteral Nutrition Associated Cholestasis
Primary: Number of Participants With Parenteral Nutrition Associated Cholestasis — 6; 6 Participants — p=0.94
Cholestasis · Primary Sclerosing Cholangitis · Primary Biliary Cirrhosis
Primary: Percentage of Participants in Whom we Could Quantify Hepatic Transport of 11C-CSar — 100; 100 percentage of participants
Postcholecystectomy Syndrome
Primary: the Number of the Participants Have Postoperative RUQ Pain — 3; 9 number of participants
Alagille Syndrome
Primary: Change From MRX Baseline to Week 48 in Fasting sBA Levels — 261.96; 128.32 μmol/L — p=0.0012
Primary Biliary Cirrhosis
Primary: DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Versus Placebo — 47; 10 percentage of participants — p=<0.0001