Search for a trial

Acute Nutritional Ketosis in VLCAD Deficiency: testing the metabolic basis for therapeutic use

To test if acute NK boosts muscle mitochondrial function in vivo in patients with VLCADD in order to establish a rational basis for therapeutic use in this disorder. A secondary objective is to gather data to test the working hypothesis that the…

A Study to Evaluate the Efficacy and Safety of GFT505 once daily on Steatohepatitis in Patients with Non-Alcoholic Steatohepatitis (NASH).
A Multicentre, Randomized, Double Blind, Placebo-Controlled study, with an adaptive design to allow for initial GFT505 80mg dosing versus placebo, followed by a second phase including GFT505 120mg dose, after review of 6-month safety analysis of the 80mg data on at least 50% of patients.

The primary objectives of this study are: - To evaluate the efficacy of GFT505 80mg and GFT505 120mg once daily (according to adaptive design, after 6-month interim safety analysis of the 80mg data on at least 50% of patients) for 52 weeks versus…

A Randomized, Controlled, Open-label, Multicenter, Phase IIb
Safety and Efficacy Study of HGT-1410 (Recombinant Human
Heparan N Sulfatase) Administration via an Intrathecal Drug
Delivery Device in Pediatric Patients with Early Stage
Mucopolysaccharidosis Type IIIA Disease

The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…

A Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Varying Doses and Dose Regimens of Evinacumab in Patients with Persistent Hypercholesterolemia Despite Maximally Tolerated Lipid Modifying Therapy

The primary objective of the study is to evaluate the reduction of low density lipoprotein cholesterol (LDL-C) by evinacumab in comparison to placebo after 16 weeks in patients with primary hypercholesterolemia (heterozygous familial…

Non-invasive rapid assessment of non-alcoholic fatty liver
disease (NAFLD) using Magnetic Resonance Imaging with LiverMultiScan (RADIcAL)

Primary objective: To investigate whether the introduction of LiverMultiScan as a standardised diagnostic test for liver disease can prove a cost effective method in different EU territories.Secondary Objectives:- To investigate patient satisfaction…

A Double-Blind, Placebo-Controlled, Cross-over Phase II Study to Evaluate the Effect of a 6-week Elafibranor (120mg) treatment administered once daily on hepatic lipid composition in subjects with Nonalcoholic Fatty Liver (NAFL).

Primary Objective * To evaluate the effect of a 6-week treatment with elafibranor versus placebo on hepatic lipid composition in subjects with a fatty liver.Secondary objectives * To evaluate the between treatment difference (elafibranor 120 mg/d vs…

An Open-Label Extension Study of the Safety of Relacorilant (CORT125134) in the Treatment of the Signs and Symptoms of Endogenous Cushing Syndrome

Primary Objectives: Safety Assessments: Effect of Administration of Relacorilant on: • Incidence of TEAEs (assessed monthly): TEAEs, SAEs, treatment-related TEAEs, TEAEs leading to early discontinuation of study treatment• Clinical laboratory tests…