12 results
The aims of this study are to assess the additional value of NBI in comparison to white-light endoscopy (WLE) for the detection and classification of HPs, SAs, MPs and adenomas in patients with HPS.
To evaluate the effects of short term supplementation of Phe to levels comparable to levels observed in adult patients who fully discontinued their diet on neuropsychological functions and wellbeing of adult patients with PKU.
To investigate whether treatment with candesartan (ARB) reduce 24-hours systolic BP and aortic stiffness more effectively than treatment with metoprolol (beta-blocker) in patients with repaired CoA and late hypertension.
The objectives of the study are to evaluate the following in patients with a documented history of EPP:•determine whether CUV1647 can reduce the number of phototoxic reactions in patients with EPP•determine whether CUV1647 can reduce the severity of…
The objective of the study is to investigate the effect of PEEP ventilation with or without a recruitment maneuver on right ventricular afterload in infants undergoing cardiac surgery.
To prospectively compare NBI with HRE for the detection and differentiation of polyps in HPS in a large multicentre HPS cohort (n~125).
Part A:To evaluate the safety and tolerability of increasing doses of VX-770 up to 450 mg (q12h) in healthy male subjects.Part B:To determine if therapeutic or supratherapeutic systemic exposure to multiple doses of VX 770 prolongs the mean…
The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…
The primary objective of this study is to show bioequivalence between nitisinone oral suspension and nitisinone capsules.Secondary objectives of this study are:- To assess the effect of food on the bioavailability of nitisinone oral suspension.- To…
The study will consist of three parts, part A, B and C. The main purpose of the study is to investigate to what extent VX-983 is safe and tolerated. In addition, it will be investigated how quickly and to what extent VX-983 is absorbed and…
Primary objective:• Assess the pharmacokinetics (PK) of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary VWDSecundary objectives:• Compare the…
Investigate the effect of adjuvant metformin therapy on insulin need and on glycaemic control in CFRD patients.