1191 results
The aim of this study is to evaluate an intervention aimed at improving family disclosure of inherited cancer risks by 1) supporting counselees in what they experience as a necessary, but at times difficult, task and, consequently 2) provide more at…
Evaluate the effects of CER-001 - adminstered as iv infusion - on plaque size/burden in arteries, plaque inflammation in the artery wall and the rate of cholesterol transport in the body
The objective of this extension study is to find out the potential long-term benefits and safety of tolvaptan. During this study all participants will receive tolvaptan.PRIMARY OBJECTIVE is to demonstrate whether tolvaptan modifies ADPKD progression…
* To study the relationship between inflammation on MRI and syndesmophyte formation with very detailedimaging methods: radiography, computed tomography (CT), 3 and 7 Tesla MRI. * To examine the time order of pathologic changes like inflammation,…
The primary purpose of the study is to investigate whether the new 20 mg capsule of Orfadin® (nitisinone) has a similar bioavailability in the body (gives the same concentration in the blood) as two of the marketed capsules of 10 mg. The secundary…
Objectives: 1. To investigate the types of antitrombin deficiency in these families2. To find the mutations in our families/patients with antitrombin deficiency.3. To establish the risk of VTE due to these mutations and subtypes.(comparing…
To evaluate the long-term safety, tolerability and efficacy of ArikaceTM 590 mg for a maximum 12 cycles administered once daily, where each cycle consists of 28 days on treatment followed by 28 days off treatment.
We aim to increase patient compliance by extending the existing treatment protocol of (oral or intravesical) anticholinergics plus application of CIC with the addition of bladder pressure measurement performed at home during CIC plus access to an…
Primary: To compare the reduction in frequency of partial-onset seizures on each of two trough ranges of everolimus (3-7 ng/mL and 9-15 ng/mL) versus placebo in patients with TSC who are taking one to three AEDs. Key secondary: Ability to completely…
- To assess the burden of disease of congenital CMV infection in the Netherlands at the age of 5 to 6 years through the assessment of the occurrence of sensorineural hearing loss due to congenital CMV infection.- To establish the burden of disease…
The main objective is to improve exercise tolerance. Secondary objectives are: 1) improvement of motor function development, daily physical activity, quality of life, self perception of motor competence, and participation; 2) evaluation of cost…
Primary objective: To investigate whether pharmacokinetics of the recommended dose of inhaled tobramycin, defined as serum tobramycin Area Under the Curve (AUC0-24hr), with the I-neb (75 mg) is equivalent to the PariLCPlus (300 mg) nebuliser in…
The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients (no prior exposure to factor products or any blood products) of less than 6 years of age.
The main objectives of this study are feeding techniques and skills and weight gain. Secondary objectives are NS feeding *feeding sequelae*, extent of the cleft, associated malformations, upper respiratory infections / pneumonia, medication (child…
The primary objective of the study is to demonstrate the reduction of LDL-C by REGN727 as add-on therapy to stable, maximally-tolerated dialy statin therapy with or without other LMT in comparison with placebo after 24 weeks of treatment in patients…
The following research questions were formulated:• Does blood group P status (presence or absence of the 42C>T-SNP) correlate to GB3 content on fibroblasts and white blood cells of healthy controls?• Does blood group P status correlate to (…
The family with the TRα2 premature stop enables us to investigate the biological role of the orphan wild-type TRα2 and the consequences of its dysfunction. The primary objectives of this proposal are (i) to search for a biological function of TRα2…
This study is aimed at determining pharmacokinetics and pharmacodynamics of medications routinely used at the Erasmus Medical Centre in pediatric cardiac surgery during and after cardiopulmonary bypass. The goal is to be able to formulate evidence…
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein (LDL) cholesterol (LDL-C) by REGN727 in comparison with ezetimibe (EZE) 10 mg PO QD after 24 weeks in patients with primary hypercholesterolemia (…
To gain a better insight into the genetic factors that play a role in the etiology of the thoracic aortic aneurysm with the following specific aims:1. Study of specific geneitc factos that play a role in the pathogenesis of both syndromic and non-…