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Bèta-TCP (bèta-tricalcium phosphate) as a bone substitute in patients undergoing repair of the alveolar cleft in CLP(Cleft Lip Palate) patients.

This current study aims to investigate the use of Curios bèta-TCP, a synthetic tricalcium phosphate for repair of the alveolar cleft in patients with clefts of lip, alveolus and palate.The specific properties of Progentix TCP result in optimal and…

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.

An Efficacy and 2-Year Safety Study of Open-label Rosuvastatin in Children and Adolescents (aged from 6 to less than 18 years) with Familial Hypercholesterolaemia

Primary Objectives * To assess the efficacy of rosuvastatin in paediatric patients with familial hypercholesterolaemia. * To establish long-term safety, tolerability and efficacy of rosuvastatin in paediatric patients with familial…

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.

Spatio-Temporal Image Correlation; the Key to fetal EchocaRdiography
Part III signs of decompensation in growth-restricted fetuses

The focus of this study is to get more insight in the pathophysiology of IUGR. The main goal is to investigate the applicability of STIC in determining fetal cardiac function in IUGR.

Coronary artery calcification measured with multi detector-row computed tomography in patients with hemophilia A

To investigate whether patients with hemophilia A develop less coronary artery atherosclerosis as compared to non-hemophilic males.

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…

Clinical and molecular genetic aspects of idiopathic epilepsies

(1) Identification of novel loci and genes in which mutations are responsible for the development of inherited epileptic syndromes. (2) Establishing genotype-phenotype correlations; defining the yield of mutation screenings in specific patient…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study
Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher
Disease Type 1

The primary objective of this study is to confirm the efficacy and safety of Genz 112638 after 39 weeks of treatment in patients with Gaucher disease type 1.The secondary objective of this study is to determine the long term efficacy, safety, and…

Phase 2a, Double-Blind, Placebo-Controlled, 2-Period, 2-Treatment, Crossover Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Multiple Oral Doses of XPF-001 (400 mg bid) in Patients with Inherited Erythromelalgia

• To evaluate the safety and efficacy of multiple doses of XPF-001 (400 mg bid) for relief of pain in patients with IEM.• To evaluate the efficacy of multiple doses of XPF-001 (400 mg bid) for relief of vasomotor signs in patient with IEM.• To…

Topical rapamycin to treat fibrofolliculomas in Birt-Hogg-Dubé syndrome

Primary Objective: To determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary Objectives: Safety, formula acceptability and…

Effect of short-term, high protein intake on whole-body protein synthesis and breakdown and stress response in infants after cardiac surgery

Primary: To measure the effect of a high protein diet on whole body protein synthesis and -breakdown, as well as on net whole body protein balance and production of urea in children after cardiac surgerySecondary: To measure the effect of a high…

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…

Visualizing VEGF producing lesions in Von Hippel-Lindau disease

The primary objective is to determine if disease associated lesions in patients with VHLD can be visualized with 89Zr-bevacizumab PET scans. Secondary objectives are to explore if 89Zr-bevacizumab PET imaging can differentiate progressive from non-…

Coagulation in Lamin A-C mutations

To establish whether carriers of LMNA mutations have a hypercoagulable state compared to non-affected family members.

Genetics of pelvic organ prolapse; identification of specific gene defects in patients and their family members.

Genetic analysis of the familial inheritance pattern of the COL3A1 2209G>A polymorphism.

A systematic metabolic approach to the evaluation of nutrition in Rett syndrome according to the cardiorespiratory phenotype in Dutch Rett girls.

The aims of this pilot study are to describe the nutritional status of a well-defined group of Dutch RTT girls with complete clinical, molecular and neurophysiological work-up (full description is presented in the section *study population*) and to…

Spatio-Temporal Image Correlation; the Key to fetal EchocaRdiography
Part I Examination of the cardiac anatomy of the normal fetus.

We will investigate the feasibility of using STIC technology to evaluate the normal fetal cardiac anatomy and morphology in different stages of gestation. Further we aim at making normograms of different intracardiac parameters in relation to…

Does classic galactosemia affect male fertility?

Comparing fertilty of male galactosemia patients to a control group

A randomized, double-blind, controlled phase III study of Stimuvax® (L-BLP25 or BLP25 liposome vaccine) in combination with hormonal treatment versus hormonal treatment alone for first-line therapy of post-menopausal women with estrogen
receptor (ER)-positive and/or progesterone receptor (PgR)-positive, inoperable locally advanced, recurrent, or metastatic breast cancer

PrimaryDemonstration of superior efficacy (as measured by PFS time) of L-BLP25 in combination with hormonal treatment (HT) over placebo plus HT, when used for first-line therapy of hormone receptor-positive (ER+ and/or PgR+), inoperable locally…