30 closest matches · ranked by relevance
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 · Alagille Syndrome · Cholestatic Liver Disease
Primary: Frequency of Treatment-emergent Adverse Events [TEAEs] — 16; 10; 4; 3 Participants
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
Primary: Change From Baseline in Serum Bile Acids — -104.00; -139.84; -57.97 micromole per liter (µmol/L)
Primary Biliary Cirrhosis
Primary: Serum Level of Alkaline Phosphatase — 351; 175 U/L — p=<0.0001
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 (PFIC)
Primary: Change From Baseline to Endpoint (Week 13) in Fasting sBA Level — -23; 18; -38 umol/L
Primary Biliary Cholangitis · Cardiovascular Abnormalities
Primary: The Incidence of Cardiac Events — 0; 0; 0; 0 Participants — p=0.001
Infant Development · Gut Microbiome · Food Protein Induced Allergic Proctocolitis
Primary: Changes to Gut Microbiome Composition — 42.816; -12.413 Change in %B.infantis reads (Day 28-BL) — p=0.154
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
Primary: Number of Participants With Treatment Emergent Adverse Events — 21; 10; 16; 15 Participants
Primary Biliary Cirrhosis
Primary: Change in Serum Alkaline Phosphatase Levels. — -57.3 U/L — p=0.001
Hepatic Impairment
Primary: Maximum Plasma Concentration (Cmax) of PF-05221304 — 1220; 1591; 1433; 1592 Nanogram per milliliter (ng/mL)
Nonalcoholic Fatty Liver Disease · Nonalcoholic Steatohepatitis
Primary: Percent Change From Baseline in Liver Fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI- PDFF) at Week 16 — -7.2; -17.1; -49.9; -55.9 Percent change
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
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
Familial Partial Lipodystrophy
Primary: Change in the Liver Triglycerides (TG). — 13.2; 6.4; 12.3; 10.6 percentage of liver triglycerides
Autoimmune Hepatitis
Primary: Safety/Tolerability Measured by Clinical Laboratory Tests, Metabolics, Cytokines, Anti-PIF Antibody, Periodic Physical Examination, Including Vital Signs Measurements…
Primary Biliary Cirrhosis
Primary: Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration — -0.38 mmol/L
Non-alcoholic Fatty Liver Disease
Primary: Percent Change From Baseline in Whole Liver Fat at Week 6 — -7.97; 2.84; -25.43 Percent Change — p=0.1654
Hepatitis C, Chronic
Primary: SVR12: Plasma HCV RNA Level Less Than 25 IU/mL at 12 Weeks After End of Treatment (EOT) — 61.1; 52.9 Percentage of participants
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
Familial Chylomicronemia Syndrome
Primary: Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10 — -85.7; -89.5; 38.8 Percent change — p=0.0022
Primary Sclerosing Cholangitis
Primary: Percentage Change From Baseline Through Week 24 in Serum Alkaline Phosphatase (ALP) — -4.5 percentage change in ALP
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Familial Partial Lipodystrophy · Hypertriglyceridemia · Fatty Liver
Primary: Change in Fasting Serum Triglyceride (at 12 Weeks) — -0.44; -20.27 percent change — p=0.517
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
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
Hepatic Impairment
Primary: Maximum Observed Plasma Concentration (Cmax) for PF-04965842 — 1352; 1276; 1426 nanogram per milliliter (ng/mL)
Hypoplasminogenemia · Congenital Plasminogen Deficiency
Primary: Overall Clinical Success in Number and Size of Lesions as Measured by Photographic or Other Imaging Modality Depending on the Organ System Affected or Change in Affected…