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A Randomized, Controlled, Open-label, Multicenter, Phase IIb
Safety and Efficacy Study of HGT-1410 (Recombinant Human
Heparan N Sulfatase) Administration via an Intrathecal Drug
Delivery Device in Pediatric Patients with Early Stage
Mucopolysaccharidosis Type IIIA Disease

The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…

A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis.

The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…

Brown adipose tissue activity and energy metabolism in cachexia induced by cancer or chronic disease

To study BAT activity and energy metabolism in patients with cachexia induced by cancer or chronic disease.

Intestinal production of Imidazole Propionate in healthy and obese subjects with or without DM2;
a pilot trial

To study the pharmacokinetics of non-reactive labelled histidine (L-HISTIDINE:HCL:H2O (D5, 98%; 15N3,98%) and the effect on intestinal imidazole propionate (IP)production in small intestine vs colon in healthy controls, metabolic syndrome and DM2…

Whole-body MRI in Gaucher disease; monitoring disease activity, early detection of complications and treatment decision-making

The objective of the study is to investigate the applicability of the whole body MRI protocol in Gaucher disease. To do so, we will investigate:1. If whole-body MRI ironmeasurements represent the distribution and total amoount of Gauchercells in the…

Selecting Outcome measures for MITOchondrial disease in children: an explorative study

To study feasibility, reliability and validity of a specific set of outcome measures in children with mitochondrial myopathy.

Acute Nutritional Ketosis in VLCAD Deficiency: testing the metabolic basis for therapeutic use

To test if acute NK boosts muscle mitochondrial function in vivo in patients with VLCADD in order to establish a rational basis for therapeutic use in this disorder. A secondary objective is to gather data to test the working hypothesis that the…

Prevent CardioKids

The aim of Prevent CardioKids is to evaluate to what extend disturbed vascular function markers are present in children of different weight categories and whether these are age dependent. Also differences * and relationships between * vascular…

Adipokines gene expression in visceral adipose tissue in patients with esophageal adenocarcinoma

The purpose of this study is to investigate the role of adipokines-secretion in visceral adipose tissue and its influence on the risk for esophageal adenocarcinoma. Examination of adipose tissue may provide important insight into mechanistic links…

Acute effects of capsaicin on energy expenditure, fat oxidation and satiety in negative energy balance

To investigate the acute effects of capsaicin on energy expenditure, substrate oxidation, appetite profile and ad libitum energy intake during negative energy balance.

Probiotic functionality of several probiotic supplements during gastro-intestinal transit in vivo in healthy human subjects

Primary objectiveTo measure the conversion rate of mono- and disaccharides into oligosaccharides and mannitol in the digestive tract by the action of food grade micro-organisms Secondary objectivesTo measure pH of gastric and duodenal fluids at…

Human Brown Adipose tissue perfusion dynamics. The use of functional MRI.

To study the application of fMRI for the detection of BAT perfusion.

Nutrient sensing in response to starvation in obese and lean individuals

1. To compare the effects of fasting on energy sensing machinery in muscle of obese vs normal weight subjects2. To compare neuronal activity in the brain in response to fasting in obese vs normal weight humans3. To compare the activity of the HPA…

Macrophage infiltration and adipocytokine expression in subcutaneous and visceral fat tissue in patients with Cushing's syndrome.

To describe the differences in macrophage infiltration and mRNA expression of adipocytokines in subcutaneous, omental and perirenal fat tissue in Cushing's syndrome compared to healthy controls.

Effects of beef protein consumption on energy intake - The protein leverage hypothesis

To determine ad libitum daily energy intake in response to protein/carbohydrate and fat ratio over 12 consecutive days, and in relation to age, gender, BMI and FTO gene alleles.

A Double-blind, Randomized, Placebo and Ezetimibe Controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 on LDL-C in Combination With Statin Therapy in Subjects With Primary Hypercholesterolemia and Mixed Dyslipidemia (AMG 145 20110115)

Primary: To evaluate the effect of 12 weeks of subcutaneous (SC) AMG 145 every-2-weeks (Q2W) and every-4-weeks (Q4W), compared with placebo, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C).Secondary objectives: Safety…

Natural course of Niemann-Pick disease type C in the Netherlands

The first aim of this study is to extend clinical knowledge on NPC. This has several reasons:1) The heterogeneous clinical presentation of NPC makes extensive clinical knowledge amongst physicians a prerequisite in preventing diagnostic delay.…

Dietary protein requirements and caloric over-consumption on unbalanced diets

To determine ad libitum daily energy intake, energy balance and appetite profile in response to protein/carbohydrate and fat ratio over 12 consecutive days, and in relation to age, gender, BMI and FTO polymorphisms.

Effect of Fecal microbiotA Transplantation combined with MEditerranean Diet on insulin sensitivity in subjects with metabolic syndrome

To study the effect of a Mediterranean diet (MD) followed by lean donor fecal microbiota transplantation (FMT) versus the prescription of Mediterranean diet (MD) followed by autologous (own) FMT in male subjects with metabolic syndrome on peripheral…

The impact of short-term bed-rest on skeletal muscle mass and insulin sensitivity in healthy, young men

To investigate the impact of 7 days of bed-rest on skeletal muscle mass, strength and whole-body insulin sensitivity in healthy, young men.