8 results
Primary Objective* To compare the effect of a 30-week course of mavacamten with placebo on clinical response comprising of exercise capacity and clinical symptoms in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)…
DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild hemophilia A.
Primary objectives:To assess the proportion of non-severe hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours after the start of combination treatment, without adding off-…
To investigate whether peri-operative dosing using a population-based pharmacokinetic model (non-linear mixed effect modelling) in hemophilia patients leads to a significant reduction in clotting factor consumption in comparison to the standard…
To investigate if subjects with an increased risk of developing spondyloarthritis have evidence of bone formation on [18F] Fluoride PET-CT.
The aim is to investigate the therapeutic effects of Secukinumab on bone formation in SpA patients by using [18F]Fluoride PET-CT imaging, and to investigate cellular and molecular pathology in bone formation.
primary objective:To assess the long-term safety and tolerability of mavacamten in participants with hypertrophic cardiomyopathy (HCM) previously enrolled in 1 of 2 placebo-controlled trials: MAVERICK-HCM (MYK-461-006) for non-obstructive HCM (nHCM…
Primary Objectives: • To assess the efficacy of a 48-week course of mavacamten compared to placebo on patient- reported health status (symptoms and physical limitations)• To assess the efficacy of a 48-week course of mavacamten compared to placebo…
The primary objective of this proof-of-concept study is to investigate the feasibility of whole body [18F]Fluoride PET-CT scans to detect axial and peripheral neew bone formation in PsA patients taht are at risk to develop clinically manifest PsA.…