37 results
Primary Objectives*To evaluate the safety and tolerability of multiple oral doses of GLPG1837 in subjects with CF and at least one copy of the S1251N mutation.Secondary Objectives*To assess changes in sweat chloride from baseline (Day 1) as the…
To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS and to compare the effects of different doses and frequencies of oxytocin administration
Primary objective of this study is to evaluate the clinical effect of a long term treatment (8 weeks) with oral B2-agonists in CF patients with residual CFTR function, especially on lung function (spirometry and airway resistance). Secondary…
Primary objective: To determine the safety of once daily inhalation of the recommended daily dose of tobramycin with the Akita® and the PARI-LC® Plus nebulizer in patients with CF. Systemic absorption can be used as surrogate parameter for safety.…
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…
Primary objective of this pilot study is to evaluate the in vivo effect of a B2-agonists in CF patients with residual CFTR function, using dosages which are clinically used in patients with asthma. Secondary objective is to evaluate the difference…
* To examine how multiple oral doses of the test compound (an experimental medication) will be taken up by the body, metabolized and excreted by the body in combination with the oral contraception pill which consists of ethinyl-estradiol and…
To discover human genes which are likely to confer susceptibility for recurrent respiratory papillomatosis
1. What are the effects of cinacalcet on clinical and biochemical parameters, including bone turnover markers, in patients with primary hyperparathyroidism due to a MEN-I mutation?2. What are the effects of cinacalcet on bone mineral density and…
The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…
The aim of this study is to objectify nipple-areola complex sensitivity in women who underwent unilateral or bilateral prophylactic NSM in Erasmus MC-Daniel den Hoed Cancer Center and their satisfaction with this operation, compared to healthy women…
To explore how HD mutation carriers experience suicidal ideation, plans, or previous suicide attempts (from now on jointly referred to as suicidality), how they cope with suicidality, and which ideas and wishes HD mutation carries have on how…
PrimaryTo evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:* Incidence and severity of adverse events and serious adverse events* Changes in vital signs, laboratory assessments, and ECGs*…
Primary:* To determine the absolute oral bioavailability and further characterize the PK of RO5186582 using a stable isotope technique.* To explore the routes and rates of elimination of [14C] labelled RO5186582.Secondary:* To identify and quantify…
The primary objective of the study is to examine the feasibility of measurements, program design and implementation adherence of a cardiovascular exercise program in persons with Down*s syndrome suffering from Alzheimer*s disease. Secondary…
The primary objective is to determine the pharmacokinetic characteristics of galantamine after 2 single oral doses in children with Down syndrome. Secondary objectives are to evaluate safety and tolerability.
Primary objective:To compare the change in small airways obstruction (FEF75%) in patients with CF when inhaling one ampule of inhaled tobramycin with the Akita® compared to standard of treatment (twice daily nebulization of one ampule using standard…
Please refer to protocol, section 1.2 "Rationale"
Main objective: to assess the effects of PEG treatment on bile acid homeostasis in cystic fibrosis patients. Secondary objective: to assess the effects of PEG treatment on gastrointestinal symptoms and quality of life, and on gut health-related…
The objective of this study is to examine the effect of riluzole on the glutamate/GABA balance in the brain in patients with 22q11.2DS. The secondary objective is to examine the effects of riluzole on psychotic symptoms and cognitive functioning. In…