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Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-Toxin-A (Botox): a pilot study.

- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function?- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an…

Long-term developmental outcomes of early detection and intervention in Permanent Childhood Hearing Impairment

Aim1. To investigate the long-term effects of early detection and intervention of hearing loss on the development of children with congenital bilateral hearing impairment. 2. To define factors in early childhood that are able to predict…

A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Human Antihaemophilic Factor VIII (rFVIII; INN: octocog alfa) in Subjects with Hemophilia A, and a Repeat PK, Safety and Efficacy Study

The study objectives are to determine the incidence rate of FVIII inhibitors, frequency of adverse events (AEs), and serious adverse events (SAEs) associated with the use of CSL627, to evaluate the PK of 50 IU/kg CSL627, and to evaluate the efficacy…

Propofol during Minimal Invasive Surfactant Evaluation Study

The primary objective of this study is to compare the level of stress and comfort of preterm infants when sedation is given during the MIST procedure compared to no sedation.

A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients with Severe Hemophilia A

Primary ObjectiveThe primary objective is to compare the annualized rates of bleeding episodes (ABR)between subjects receiving a prophylactic regimen of BAX 855 with an on-demandtreatment regimen.Secondary ObjectivesThe key secondary objective is to…

A Phase III, randomised, double blind, placebo controlled, parallel group study, to evaluate the safety and efficacy of subcutaneous implants of afamelanotide (16 mg) in patients suffering from polymorphic light eruption (PLE).

Primary Objective - To determine if afamelanotide can reduce the severity of PLE related pruritis.Secondary Objectives- To determine if afamelanotide can reduce the frequency of PLE episodes;- To determine if afamelanotide can reduce the duration of…

A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP).

Primary objective: - To determine whether afamelanotide can reduce the severity of phototoxic reactions in patients with EPPSecondary objectives:- To determine whether afamelanotide can reduce the number of phototoxic reactions in patients with EPP…

Urethral diverticulae in laparoscopic management of high and intermediate anorectal malformation

exclusion or demonstration of a diverticulum in patients undergoing a laparoscopic pull-through

Prevention of local thromboembolic complications during continuous infusion of coagulation factor concentrates: parallel infusion of saline versus heparin and parallel infusion of saline?

To demonstrate that parallel infusion of saline added to the continous infusion of clotting factor concentrates prevents local thromboembolic complications to the same extent as heparin together with a parallel infusion of saline does.

High frequency oscillation versus conventional mechanical ventilation in newborns with congenital diaphragmatic hernia: an international randomized controlled trial

Primary objective: -to determine if there is a significant difference in the incidence of oxygen dependency at day 28 and/or death within the first 28 days of life between newborns with congenital diaphragmatic hernia treated with high frequency…

Factors that influence women*s utilization of prenatal screening for Down*s syndrome. Optimizing decision making and refining current and future screening policies by studying differences in uptake rates, between three European countries*

The striking difference in uptake of Down*s Syndrome screening between countries that otherwise show close resemblance, particularly regarding healthcare policy, social and cultural factors (including non-directiveness of counselling, autonomous and…

Exercise training in congenital heart disease: is it effective and safe and does it improve quality of life

Objectives:Medical: What is the effect of an exercise training program in children and young adults (up to 20 years of age) who have undergone treatment for ConHD (tetralogy of Fallot or Fontan procedure) on: a) aerobic fitness, b) ventricular size…

medroxyprogesterone acetate (MPA) in Familial Adenomatous Polyposis, a proof of principle study

To assess in a cohort of patients with established FAP:1 The efficacy of MPA in terms of reduction of number of colonic polyps, by means of Endoscopic Appearance of Polyposis (EAP) index.2 The effect on histological parameters and biological…

A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP)

The objectives of the study are to evaluate the following in patients with a documented history of EPP:•determine whether CUV1647 can reduce the number of phototoxic reactions in patients with EPP•determine whether CUV1647 can reduce the severity of…

Leiden's IVIg trial in Rhesus disease of the Neonate

AimTo determine whether the prophylactic use of IVIg reduces the need for ET in neonates with Rh-D hemolytic disease.

The adenosine receptor; a new pharmacological tool in the treatment of sepsis.

Does antagonism of the adenosine receptor by caffeine lead to an increased LPS-induced inflammatory reaction and an increase in (subclinical) tissuedamage?Does the C34T-polymorphism of the enzyme AMP-deaminase lead to a decreased inflammatory…

Pilot study of everolimus in the treatment of advanced malignancies in patients with Peutz-Jeghers syndrome

To determine the efficacy (as measured by objective tumor response) of single agent everolimus in the treatment of advanced malignancies or high risk polyps of patients known with PJS .

Dendritic cell vaccination in patients with Lynch Syndrome or colorectal cancer with MSI

The primary objective is the safety and feasibility of CEA/frameshift derived neopeptide loaded DC in patient with MSI-positive colorectal cancer and persons who are known to be carrier of a germline MMR-gene mutation with no signs of disease yet.…

A randomized controlled trial on the effects of periconceptional and prenatal folic acid supplementation on congenital anomalies and preterm birth

In the proposed study, two main issues will be addressed: first, it will be studied whether a high dose (4.0 mg) of FA supplementation periconceptional has an added value over the now recommended low dose of 0.4 mg in the prevention of congenital…

Improving risk assessment in consanguineous couples by means of SNP analysis

The objective of this research is to develop a method which can identify among all consanguineous couples the ones which are at high risk.