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Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, open-label, cross-over trial of different treatment regimens of oxytocin administration. Effects on eating behaviour and social behaviour.

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

A Phase 3 clinical study to determine the pharmacokinetics, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in subjects diagnosed with von Willebrand disease

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…

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…

A B2-agonist as a CFTR activator in CF

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…

A study to evaluate the bioequivalence of Orfadin capsules 20 mg compared to Orfadin capsules 10 mg. An open-label, randomized, cross-over, single-dose study in healthy volunteers.

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…

Bioequivalence of Orfadin suspension 4 mg/mL compared to Orfadin capsules 10 mg, and the effect of food on the bioavailability of the suspension.
An open-label, randomized, cross-over, single-dose study in healthy volunteers.

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…

Endoscopic surveillance using narrow-band imaging in patients with hyperplastic polyposis syndrome (HPS): A multicentre cohort study

To prospectively compare NBI with HRE for the detection and differentiation of polyps in HPS in a large multicentre HPS cohort (n~125).

Clinical study of the effect of PEEP ventilation on right ventricular afterload in pediatric patients after cardiac surgery.

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.

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.

Colonoscopic surveillance using narrow-band imaging in patients with hyperplastic polyposis syndrome (HPS)

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.

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…

SPACE trial
SMA and Pyridostigmine in Adults and Children; Efficacy trial
Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4

to investigate the effect and efficacy of pyridostigmine on muscle strength and fatiquabillity in patients with SMA.

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 (…

High intensity interval training in patients with tetralogy of Fallot

Primary Objective: To examine the effect of an online based exercise training protocol on ventriculo-arterial coupling , as assessed by pulse wave velocity and wave reflection patterns in the aorta and pulmonary artery in patients operated for…

An open-label, single-sequence cross-over drug-drug interaction study to evaluate the effect of single and multiple oral doses of PHA-022121 on the pharmacokinetics of selective CYP1A2, CYP2C19, and CYP3A4 substrates in healthy subjects

The main purpose of this study is to investigate the effect of multiple doses of PHA-022121 on how quickly and to what extent a drug cocktail (a combination of agents consisting of caffeine, omeprazole, and midazolam) is absorbed, distributed,…

Treating Leg Symptoms in Women with X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance

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…

A phase I, open-label, randomized, two period, two-way cross over study to assess the influence of a high-calorie, high fat meal on the bioavailability of a 40 mg extended release PHA 022121 oral formulation

In this study we will investigate the influence of a high-calorie, high-fat meal on the uptake, breakdown, and elimination of the study compound PHA-022121. We will also investigate how safe PHA-022121 is and how well it is tolerated when it is used…

The effect of a low carbohydrate- high fat diet on clinical symptoms and gut microbiome in m.3243A>G related mitochondrial disease

To explore the effect of a LCHF diet on clinical symptoms and gut microbiome in MD patients due to the m.3243A>G mutation in mitochondrial DNA.

A Phase I, two parallel arms, open label, randomized, two period, two-way cross-over study to assess the steady-state pharmacokinetics of a 40 mg PHA-022121 XR tablet administered once daily with the steady-state pharmacokinetics of two 10 mg PHA-022121 IR capsules administered twice daily (Arm 1) and to assess the steady-state pharmacokinetics of 40 mg PHA-022121 XR tablet administered twice daily with the steady-state pharmacokinetics of four 10 mg PHA- 022121 IR capsules administered twice da

In this study we will investigate how quickly and to what extent a formulation of the study compound PHA-022121 is absorbed, transported, and eliminated from the body. There are two compositions of the study compound: one with an extended release…