158 results
Primairy Objective* To test whether 5-ASA reduces the occurrence of colonic benign or malignant neoplasia compared to placebo in Lynch syndrome (LS) patients as detected by any colonoscopy until the end of study.Secondary Objectives* To test wheter…
Primary Study Objective: The primary objective of this study is to assess the efficacy of laquinimod 0.5 mg and 1.0 mg qd in patients with HD after 12 months of treatment using the UHDRS-TMS.Secondary Study Objectives:• To assess the effect of…
To evaluate the effects of intranasal oxytocin versus placebo on social behaviour and also on eating behaviour in children with PWS.
This study aims to demonstrate the clinical efficacy of patisiran and to establish the safety of chronic dosing in ATTR patients with FAP.The primary objective of the study is to determine the efficacy of patisiran by evaluating the difference…
The primary objective of the study is Study to determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD)
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
Primary ObjectiveTo determine the safety and tolerability of multipledoses of PF-04447943 Secondary ObjectiveTo characterize the PK of PF-04447943 in plasma following oral administrationExploratory ObjectivesTo evaluate biomarkers that may be…
The purpose of Part A is to investigate how safe the study compound VX-152 is and how well the study compound is tolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminated from the body (…
The purpose of the study is to investigate how safe GLPG2737 is and how well GLPG2737 is tolerated. It will also be investigated how quickly and to what extent GLPG2737 is absorbed into, distributed in, and eliminated from the body (this is called…
To evaluate the efficacy of VX-661 in combination with ivacaftor and ivacaftormonotherapy through 8 weeks of treatment in subjects with cystic fibrosis (CF)who are heterozygous for the F508del mutation on the CF transmembraneconductance regulator (…
Primary: The primary objective of the randomized treatment epoch and for the overall study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease…
The purpose of Part A is to investigate how safe the study compound VX-440 is and how well the study compound istolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminatedfrom the body (this…
The primary objective of this study is to compare the efficacy of eprotirome 50 microgram and eprotirome 100 micorgram versus placebo in terms of the percent change in LDL-C from baseline to Week 12 in HeFH patients with CAD, or who are at high risk…
Primary: to assess efficacy of subcutaneous 6 mg/kg GSK2402968 versus placebo administered over 48 weeks in ambulant subjects with DMD. Secondary: Safety, tolerability, PK, quality of life.
Primary Objective: To determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary Objectives: Safety, formula acceptability and…
The primary objective of this study is to confirm the efficacy and safety of Genz 112638 after 39 weeks of treatment in patients with Gaucher disease type 1.The secondary objective of this study is to determine the long term efficacy, safety, and…
This study will evaluate the efficacy and safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). This study will compare the effects of 24 weeks of infusions of BMN 110 at…
- To evaluate the safety and toleration of SPC5001 in healthy subjects and in subjects with FH.- To assess the lipid lowering effect of SPC5001 in healthy subjects and in subjects with FH.- To assess the pharmacokinetics of SPC5001 in healthy…
To assess the efficacy of bosentan in neonates with persistent pulmonary hypertension of the newborn (PPHN) who are in need of continued inhaled nitric oxide (iNO) after at least 4 hours of continuous iNO treatment and to evaluate the…
Primary: To evaluate the effect of 12 weeks of subcutaneous (SC) AMG 145, compared with placebo, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in subjects with heterozygous familial hypercholesterolemia.Secondary…