19 results
Primary objective is to objectively investigate the therapeutic potential of Ivacaftor in Dutch CF patients carrying a mutation associated with residual CFTR function. A secondary objective is to evaluate the correlations between individual…
Primary objective: To show that the variability of whole blood total and unbound plasma tacrolimus concentrations during the first 6 days post transplantation is larger than the variation of tacrolimus concentrations in stable clinical situation.…
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.
We hypothesize that hypertonic saline nebulizations improve respiratory symptoms and quality of life in PCD patients.
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…
Evaluate whether FVIII/VWF concentrates successfully induce immune tolerance in patients who have already experienced and failed immune tolerance induction with VWF-free FVIII concentrates.
To show that an individually tailored treatment of the calf muscles with BTX-A promotes the balance capacity and mobility of HSP patients by an effective reduction of spasticity while preserving muscle strength. This explorative study uses a pre-…
This study is being conducted in patients with familial hypercholesterolaemia (FH) to:1. Assess the efficacy of colesevelam added to a maximal tolerated and stable regimen of statin and ezetimibe in further decreasing the low-density lipoprotein (…
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…
The primary objective of the study is to evaluate neurocognitive function with use of Praluent after 96 weeks of treatment versus placebo.
-To assess autonomic parasympathetic and sympathetic function as well as vascular function in a population of patients with univentricular hearts palliated by the Fontan procedure and compare it with the results in healthy subjects.-To assess the…
To show a reduction in FVIII-concentrate consumption with perioperative desmopressin and FVIII concentrate combination treatment compared to FVIII concentrate monotherapy, without decreasing the effectivity of treatment.
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…
This study aims to evaluate the PK profile and IIV of drug exposure in plasma in children (aged 6-17 years) receiving (elexacaftor-)tezacaftor-ivacaftor. Secondary objectives are to evaluate the relationship between covariates and PK parameters that…
This study has been transitioned to CTIS with ID 2024-518989-27-00 check the CTIS register for the current data. Objective: Primary Aim (PHASE 1):To determine the prevalence of RLS in women with ALD.Secondary Aim (PHASE 2):To determine whether in a…
In this study, we want to investigate the effect of the change in co-medication from lumacaftor to tezacaftor on the kinetics and exposure levels of ivacaftor described by the through concentrationsPrimary Objective: - the through concentration of…
To study the effect of crizanlizumab on the hemodynamics of the cerebral vasculature (CBF and CVR)
This study has been transitioned to CTIS with ID 2024-518652-23-00 check the CTIS register for the current data. Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on…
This study has been transitioned to CTIS with ID 2024-518215-18-00 check the CTIS register for the current data. To explore safety, tolerability and efficacy of avalglucosidase alfa in previously treated alglucosidase alfa late-onset Pompe disease…