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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…

Urethral diverticulae in laparoscopic management of high and intermediate anorectal malformation

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

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.

Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD).

Primary: Dose finding for a twice daily regimen for PSZ as prophylactic treatment in children with CGD, based on the exposure to PSZ measured by PSZ trough levels.Secondary:To determine the tolerability of PSZ as prophylactic treatment in children…

Ten years of statin therapy started in childhood: efficacy, safety, compliance and tolerability in patients with familial hypercholesterolemia (AfterTen-study)

This study will evaluate the effect of approximately 10 years of pravastatin treatment on IMT in young adults with FH. Furthermore, safety parameters and psychological aspects with respect to early treatment initiation will be investigated.

Radicality of (simple) mastectomy: residual breast tissue

Riskreduction of developing breast carcinoma by means of better surgical techniques (removing all normal breast tissue).

A randomised controlled trial on the effects of an individual tailored lifestyle intervention to minimise the cardiovascular disease risk of individuals with Familial Hypercholesterolemia (FH)

The study aims at examining the effect of an individual tailored lifestyle intervention to reduce the cardiovascular disease risk in people with FH.Secundary, the study aims at promoting a healthy lifestyle (no smoking, proper nutritional pattern,…

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…

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…

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…

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.

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…

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…

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…

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.

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…

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…

An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of prenatal and/or postnatal infusion of allogenic expanded fetal mesenchymal stem cells for the treatment of severe Osteogenesis Imperfecta compared with historical and untreated prospective controls.;In the Netherlands patients will not be treated. Retrospective and prospective control patients will be included

This study has been transitioned to CTIS with ID 2023-504593-38-00 check the CTIS register for the current data. The aim of this trial is to infuse multiple doses of human 1st trimester liver-derived MSC for the treatment of severe OI to determine…