9 results
Primary Objective:To evaluate the efficacy of VX-150 for the treatment of pain caused by small fiber neuropathy.Secondary Objectives:* To evaluate the safety and tolerability of VX-150* To evaluate the pharmacokinetics (PK) of VRT-1207355 and the…
The objectives of this multi-centre, double-blind, randomized, 2-parallelgroups study are to investigate the efficacy, safety and tolerability of levetiracetam (LEV) monotherapy 15-60mg/kg/day versus valproic acid (VPA) monotherapy 10-40mg/kg/day in…
Primary:The primary objective is to determine the safety and tolerability of the combination treatment, i.e. GCb, VPA and GCV, by evaluation of adverse events (AE*s) serious adverse events (SAE*s) and all clinically significant changes in clinical…
Primary Objective: • Efficacy: To evaluate the treatment effect of seladelpar on composite biochemical improvement in cholestasis markers based on alkalinephosphatase (ALP) and total bilirubin at 12 months of treatment compared to placebo• Safety:…
Primary: • To evaluate the safety and effect on cholestasis of two seladelpar regimens (5 mg/day titrated to 10 mg/day and 10 mg/day) over 52 weeks of treatment compared to placeboKey Secondary: • To evaluate the effect of seladelpar on…
Primary:• To evaluate the long-term safety and tolerability of seladelparSecondary:• To evaluate the long-term efficacy of seladelpar• To evaluate the effect of seladelpar on patient-reported outcomes (pruritus)Exploratory• To evaluate the effect of…
Primary objective:-To confirm that the Event-Free Survival (EFS) in patients >= 3 years of age with WHO grade IV/CNS WHO grade 4 and WHO grade III/CNS WHOgrade 3 diffuse high grade gliomas as well as diffuse high grade gliomas without a…
Primary objective:To assess whether hydralazine in combination with valproate is effective in treating the symptoms of narcolepsy.Secondary objectives:- To assess the safety of treating NT1 patients with hydralazine in combination with valproate.-…
The aim of this prospective proof-of-concept study is to increase the expression of the SSTR2 in NET patients with negative or low expression to levels amenable for somatostatin analogue treatment through the use of epigenetic drugs.