212 results
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…
Period 1: The first objective, of period 1, is to compare the safety and efficacy of ABT-494 15 mg QD monotherapy, and 30 mg QD monotherapy versus weekly methotrexate (MTX) monotherapy for the treatment of signs and symptoms of rheumatoid arthritis…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
Primary ObjectiveThe primary objective for this study is to compare the clinical efficacy of weekly abatacept in combination with methotrexate to methotrexate alone in achieving Remission, defined as SDAI less than or equal to 3, at Week 24.…
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
The primary goals of this study are to: i. Identify genetic variations responsible for PLP across the whole genomeMore than 90% of SNPs are found in introns or intergenic regions and most of them are probably of little functional consequence.…
1. To identify genetic variants associated with an increased risk of ASD using different genetic models (dominant, recessive, interaction).2. To determine the relationship between different genetic variants and the associated neuropsychiatric ASD…
The objectives of this study is to determine the recovery and survival of platelet concentrates; in the first phase, a comparison will be made for platelet concentrates in plasma stored for 2-3 days versus those stored for 6-7 days; in the second…
(i) identification and cellular characteristics of red blood cells from patients with altered ion homeostasis and delivery of novel and easy-to-use diagnostics for rare anemia*s, for patients with: a) a known primary defect in the ion channels of…
Primary:2. To investigate the prevalence of vulnerable plaques, i.e. plaques with intraplaque hemorrhage on MRI, in individuals with type 2 diabetes, impaired and normal glucose tolerance and a minimal plaque thickness of 2mm on ultrasonography. To…
Collection of skin tissue and blood samples from patients with mild to moderate psoriasis for efficacy testing of a new compound in a humanized mouse model.
To examine the motor cortical control of antagonist muscle function in younger and older adults
To identify predictors for the different treatment options for obesity (1. combined lifestyl intervention plus, 2. E-health treatment, 3. bariatric surgery; inclusion for treatment options 1 and 2 takes place in the EMC, inclusion for the treatment…
To evaluate the safety of an autologous cellular collagen hydrogel-based skin graft (denovoDerm) in patients with skin defects that require definitive therapeutic coverage. DenovoDerm can be transplanted together with an STSG to cover a full…
In this study, the following questions will be answered:1. Does larval therapy accelerate wound healing?2. Do single larval secretions accelerate wound healing?3. Is the bacterial load in the wound reduced during larval therapy or is the microbiome…
Primary Objective: The objectives of this study are to assess lumen, scaffold, vessel dimension, malapposition and tissue coverage as assessed by quantitative angiography, intravascular optical coherence tomography and intravascular ultrasound…
The objective of this study is to demonstrate the influence of pectus excavatum on the anaerobic threshold during exercise. This could be an objective measurement of exercise intolerance. An objective measurement of exercise intolerance can later…
To further characterize the transport of phenylalanine and other large neutral amino acids across the blood-brain barrier in PKU patients with high and low brain vulnerability to elevated blood phenylalanine concentrations, we will investigate…
To demonstrate the efficacy of ponatinib administered at 2 starting doses (30 and 15 mg QD) compared to nilotinib administered at 400 mg BID in patients with CP-CML who are resistant to imatinib, as measured by MMR by 12 months
This study will investigate the effect of shear and pressure on tissue viability of human skin. Skin vitality is determined by tissue oxygenation and blood flow, measured with the 'Oxygen to See'. The focus will be on individual response…