Phase 3 N=52 Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Alagille Syndrome
Primary: Change From Baseline in Scratching Score — -1.69; -0.8 score on a scale — p=0.0012
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=27 A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
Progressive Familial Intrahepatic Cholestasis · Alagille Syndrome · Cholestatic Liver Disease
Primary: Frequency of Treatment-emergent Adverse Events [TEAEs] — 16; 10; 4; 3 Participants
Phase 2 N=37 Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
Alagille Syndrome
Primary: Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus — -1.470; -1.486; -0.620; -0.580 Score on a scale — p=0.0321
Phase 3 N=7 A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
Alagille Syndrome (ALGS)
Primary: Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22 — 17.643 micromoles per liter (mcmol/L)
Phase 2 N=20 Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
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
Phase 2 N=34 An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Alagille Syndrome
Primary: Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA) — 223.62; 157.35 μmol/L — p=0.1157
Phase 2 N=31 Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
Alagille Syndrome
Primary: Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 — -21.73; 95.55 μmol/L — p=0.0464
Phase 2 N=19 An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
Alagille Syndrome
Primary: Change From MRX Baseline to Week 48 in Fasting sBA Levels — 261.96; 128.32 μmol/L — p=0.0012
Phase 2 N=52 MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
Cholestatic Liver Disease
Primary: Incidence of Treatment-Emergent Adverse Events — 36; 12; 9; 4 Participants
Phase 2 N=12 Smith-Lemli-Opitz Syndrome and Cholic Acid
Smith-Lemli-Opitz Syndrome
Primary: Change in Plasma Cholesterol — 37.8 change (%) compared to baseline — p=0.011
Phase 2 N=54 JAK Inhibitor Treatment in AGS
Aicardi Goutieres Syndrome
Primary: Mean and Standard Deviation (SD) of the AGS Scale at 52 Weeks — 5.9 score on a scale — p=<0.0005
Phase 3 N=5 GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis
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
Phase 3 N=300 Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
Severe Familial Hypercholesterolemia
Primary: Number of Participants With Adverse Events — 94; 174; 76; 143 Participants
Phase 3 N=85 Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
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
Phase 3 N=6 Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Gaucher Disease
Primary: Number of Severe Adverse Events (SAE) — 1 events
Phase 2 N=23 Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
Smith-Lemli-Opitz Syndrome
Primary: Number of Responders — 18 Participants
Phase 2 N=23 Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
Smith-Lemli-Opitz Syndrome
Primary: Serum Cholesterol to Total Sterol Ratio — 90.82; 93.99 percent total cholesterol — p=0.002
Phase 2 N=5 Study of Gemcabene in Adults With FPLD
Familial Partial Lipodystrophy · Hypertriglyceridemia · Fatty Liver
Primary: Change in Fasting Serum Triglyceride (at 12 Weeks) — -0.44; -20.27 percent change — p=0.517
Phase 3 N=66 Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Phase 2 N=13 Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome
Smith-Lemli-Opitz Syndrome
Primary: Hyperactivity Sub-scale of the Aberrant Behavior Checklist-Community (ABC-C). — 8.6; 8.6 units on a scale — p=1.0
Phase 3 N=158 Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
Heterozygous Familial Hypercholesterolemia
Primary: Percent Change From Baseline to Week 24 in LDL-C — -6.23; -44.53 percent change — p=< 0.0001
Phase 3 N=63 Compassionate Use of Omegaven in Children
Total Parenteral Nutrition-induced Cholestasis · Cholestasis · Short Bowel Syndrome
Primary: Resolution of Cholestasis for Subjects Who Received Omegaven — 40; 10; 2 Participants
N/A N=28 Chronic Liver Disease in Urea Cycle Disorders
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants
Phase 2 N=13 N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy
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…
Phase 3 N=53 Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
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
Phase 3 N=2 Use of Omegaven for Treatment With Parenteral Nutrition Associated Liver Disease
Parenteral Nutrition-related Hepatitis
Primary: Disease Progression as Measured by Serum Levels of Hepatic Enzymes
N/A N=140 A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy
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
Phase 3 N=5 Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Gaucher Disease
Primary: Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) — 2; 0; 2 participants