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A placebo-controlled trial of insulin therapy with or without adjuvant metformin in patients with Cystic Fibrosis-Related Diabetes

Investigate the effect of adjuvant metformin therapy on insulin need and on glycaemic control in CFRD patients.

Targeting Antibiotics to Pseudomonas Aeruginosa in Small airways (TAPAS) study in patients with cystic fibrosis: pharmacokinetics (PK)

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.…

Tobramycin nebulisation with I-neb (TONI) study in children with cystic fibrosis: pharmacokinetics and 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…

Comparison between beta-adrenergic blockers and angiotensin II receptor antagonists for the treatment of late hypertension in patients with repaired aortic coarctation

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.

Targeting Antibiotics to Pseudomonas Aeruginosa in Small airways (TAPAS) study in patients with cystic fibrosis

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…

A phase 1, two-part, randomized, open-label study to evaluate the relative bioavailability and food effect of test formulations of VX-152 and Ivacaftor in healthy subjects

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 (…

A randomized, single-blinded, 3-way cross-over trial to investigate the pharmacokinetics of sustained release cysteamine bitartrate (PO-001) in healthy adult volunteers.

- To assess the pharmacokinetic profile of cysteamine base after single dose administration of the new sustained-release cysteamine bitartrate (PO-001) to healthy volunteers, in comparison with Cystagon® and Procysbi®.- To collect safety/…

High intensity interval training in patients with a right ventricle to pulmonary artery conduit

Primary Objective: To assess the efficacy, expressed by change in peak oxygen consumption obtained with cardiopulmonary exercise testing (CPET), of a HIIT program in patients with an RV-PA conduit.Secondary Objective(s): To determine predictors for…

The role of glutamate in cognition in adults with chromosome 22q11 deletion syndrome

The main objective of this (pilot) study is to investigate the role of glutamate in cognitive functioning in adults with 22q11DS using a glutamatergic challenge (riluzole )and high-field MRS. We will relate glutamate concentrations in the striatum…

A single-dose, open label, randomized, three-way cross-over study in healthy volunteers to characterize the pharmacokinetics of the 300 mg trientine capsule and to assess the effect of dissolution rate and the effect of food on the pharmacokinetics of trientine.

Primary objectives:To characterize the pharmacokinetics of the 300 mg trientine capsule with a fast dissolution profile.To assess the effect of dissolution rate on the pharmacokinetics of trientine.To assess the effect of food on the…