30 closest matches · ranked by relevance
Progressive Familial Intrahepatic Cholestasis (PFIC)
Primary: Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26…
Progressive Familial Intrahepatic Cholestasis (PFIC)
Primary: Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort — -1.718; -0.628 Score on a scale — p== 0.0063
Progressive Familial Intrahepatic Cholestasis · Alagille Syndrome · Cholestatic Liver Disease
Primary: Frequency of Treatment-emergent Adverse Events [TEAEs] — 16; 10; 4; 3 Participants
PFIC1 · PFIC2
Primary: Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid (s-BA) Concentration From Baseline to the End of Treatment or Reaching a Level <=70…
Progressive Familial Intrahepatic Cholestasis (PFIC)
Primary: Change From Baseline to Endpoint (Week 13) in Fasting sBA Level — -23; 18; -38 umol/L
Primary Sclerosing Cholangitis
Primary: Number of Participants With Treatment Emergent Adverse Events — 21; 10; 16; 15 Participants
Progressive Familial Intrahepatic Cholestasis
Primary: Change From Baseline in Serum Bile Acids — -104.00; -139.84; -57.97 micromole per liter (µmol/L)
Primary Biliary Cholangitis
Primary: Fold Change in Serum Gamma-glutamyl Transferase (GGT) — 0.74; 0.41; 0.28; 0.31 fold-change — p=0.293
Primary Biliary Cirrhosis
Primary: Serum Level of Alkaline Phosphatase — 351; 175 U/L — p=<0.0001
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
Cystic Fibrosis · Liver Fibrosis
Primary: Elastographic Value in kPa Measured by Fibroscan — 4.7 kPa
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
Primary Biliary Cirrhosis
Primary: Change in Serum Alkaline Phosphatase Levels. — -57.3 U/L — p=0.001
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
Primary Biliary Cirrhosis
Primary: The Percent Change in Serum GGT. — -4.9; -19; -8.4 percent change
Niemann-Pick Disease, Type C
Primary: Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide — 32.6; 87.9 ng/ml
Primary Sclerosing Cholangitis (PSC)
Primary: Change From Baseline in MQC on Liver Biopsy at Week 96 — -0.5; 0.5; 0.0 percentage of MQC
Nonalcoholic Fatty Liver Disease (NAFLD)
Primary: Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) — 25; 18 participants — p=0.34
Parenteral Nutrition-Associated Liver Disease
Primary: The Presence of Cholestasis at Age of 28 Days or When Full Enteral Nutrition is Achieved, Whichever is Longer. — 39; 45 participants who developed Cholestasis
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
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 Biliary Cholangitis · Cardiovascular Abnormalities
Primary: The Incidence of Cardiac Events — 0; 0; 0; 0 Participants — p=0.001
Sclerosing Cholangitis
Primary: Adverse Event Frequency — 8 Participants
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 Sclerosing Cholangitis
Primary: Percentage of Participants Experiencing Treatment-Emergent Adverse Events During the Blinded Phase — 81.8; 70.0; 100.0 percentage of participants
Primary Biliary Cholangitis
Primary: Percentage of Participants With At Least a 20% Reduction in Alkaline Phosphatase (ALP) or Normalization of ALP at Week 12 Compared to Baseline — 45.2; 46.4; 11.1…
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
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Parenteral Nutrition Associated Cholestasis
Primary: Number of Participants With Parenteral Nutrition Associated Cholestasis — 6; 6 Participants — p=0.94
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants