30 closest matches · ranked by relevance
Wilson Disease
Primary: Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) — 92.8 μg/g — p=0.1002
Wilson Disease
Primary: Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6…
Wilson Disease
Primary: Mean Daily Copper Balance: Day 1 Through Day 8 — 0.8025; 0.3062 milligrams/day
Wilson Disease
Primary: Percentage Of Participants With Normalized Concentrations Of NCC — 85.7 percentage of participants
Wilson Disease
Primary: Serum NCC Concentration — 46.5; 58.7 µg/L
Wilson's Disease
Primary: Safety and Efficacy — 37 participants
Kinky Hair Syndrome
Primary: Gross Motor Development at 36 Mos of Age or at Death (Mos) — 13.743; 2.455; 15.667 Other - months
Wilson Disease
Primary: ALT — 41.38 U/L
Pantothenate Kinase-Associated Neurodegeneration
Primary: Number of Participants With Adverse Events — 22; 42; 12; 14 Participants
Parkinson's Disease
Primary: Number of Participants With Serious Adverse Events — 0; 0; 0 Participants
Pantothenate Kinase-Associated Neurodegeneration
Primary: Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 — 1.15; 1.38; 1.14; 0.86 Events per person — p=0.50
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 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…
Pantothenate Kinase-Associated Neurodegeneration
Primary: Change in Score on Barry-Albright Dystonia Scale — 2.48; 3.99 score on a scale — p=0.0761
Parkinson's Disease
Primary: Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) — 2.1; 4.2; -0.8; 3.9 Score on a scale…
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
Huntington Disease
Primary: Safety and Tolerability of PBT2 in Patients With HD — 32; 30; 28 participants
Iron Overload · Hereditary Hemochromatosis
Primary: Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) — -389.1; -610.5; -571.6 µg/L
Hepatic Impairment
Primary: Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide — 49.00; 36.26; 35.94; 69.03 ug/mL
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Parkinson's Disease · Gaucher's Disease
Primary: Brain GSH — 55; 41; 34 percent increase from baseline
Iron Overload Due to Repeated Red Blood Cell Transfusions
Primary: The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern. — 0; 0; 0; 0…
Ovarian Neoplasms Malignant (Excl Germ Cell) · Peritoneal Carcinoma · Fallopian Tube Cancer
Primary: Number of Participants With Dose-Limiting Toxicity (DLT) — 0; 0; 0; 0 Participants
Healthy
Primary: Change From Baseline in Mean Daily Copper Balance Over 2 Weeks of Repeated Daily ALXN1840 Dosing (Over Days 4 to 15) — -0.0749; 0.6901; 0.4201 mg/day
Parkinson Disease
Primary: Glutathione (GSH) Brain Levels — 0.93; 0.99 mM
Melanoma
Primary: Overall Response Rate to Treatment of Melanoma With Disulfiram and Chelated Zinc — 0 participants
Wolfram Syndrome · Diabetes Mellitus · Optic Nerve Atrophy
Primary: Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests — 0; 0; 0; 2 Participants
Huntington Disease
Primary: Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug — 2; 1; 3; 3 Participants
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
Sickle Cell Disease
Primary: Number of Participants With Adverse Events After Start of ICL670 — 131; 49; 1; 2 participants