Search for a trial

Assessment and pharmacological correction of abnormalities in chloride (Cl-), bicarbonate (HCO3-) and mucus transport in rectal biopsies of CF patients

We aim to fill up the present gap in knowledge about HCO3- and mucus transport in native human intestine, and about possible effects of pharmacological correctors on these parameters in CF, by detailed electrophysiological and biochemical ex vivo…

Prevention of breast cancer in high risk women by monitoring microrna expression in nipple aspirate fluid.

To establish biomarkers in nipple aspirate fluid, follow them in time and link them to breast cancer development at its earliest stage in women at high risk for breast cancer. Threshold values of biomarkers will be determined that point to a…

Hemoglobin Trajectories: Genetic determinants in Donors (HETEROGENIC DONORS)

To investigate the association between genetic factors and Hb recovery after blood donation. If an association can be found, the added value of genetic factors in prediction models for Hb deferral will be assessed. Also, the association between iron…

Cross-sectional characterisation of elderly using the NeuroCart.

Main objectives:To determine differences in NeuroCart profiles in different sub-groups based on cognitive testing and CSF biomarker profiling (i.e. cognitively normal, pre-symptomatic AD, prodromal AD, MCI, mild AD).To characterize a cohort of…

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…

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…

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 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…

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 .

Urethral diverticulae in laparoscopic management of high and intermediate anorectal malformation

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

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…

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.…

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…

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…

A SINGLE-ARM, MULTICENTER PHASE IIIB CLINICAL TRIAL TO EVALUATE THE SAFETY AND TOLERABILITY OF PROPHYLACTIC EMICIZUMAB IN HEMOPHILIA A PATIENTS WITH INHIBITORS

The study will investigate the safety and efficacy of emicizumab in patients with hemophilia A with inhibitors against FVIII.

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B

The purpose of this study is to investigate the safety and efficacy of rFIXFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) guideline on clinical…

Preservation of ovarian cortex tissue in girls with Turner syndrome:
A multicenter exploratory intervention study

To investigate the occurrence of live birth in women with TS after ovarian tissue cryopreservation in childhood followed by auto transplantation in adulthood.

A comparison of Glycosade® and Uncooked Cornstarch (UCCS) for the dietary management of hepatic glycogen storage diseases (GSD)

In light of the variability in response between UCCS and Glycosade® in patients with hepatic GSD (personal communication from authors and published data (Bhattacharya et al. 2007; Correia et al. 2008; Corrado M et al, 2013; Hubert A et al, 2013));…

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A

The purpose of this study is to investigate the safety and efficacy of rFVIIIFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) guideline on the…

A Phase 3 open-label, multicenter study of the safety, efficacy, and pharmacokinetics of intravenous recombinant coagulation Factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated pediatric patients <12 years of age with severe hemophilia A

To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A.