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A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)

Protocol version Amendment 1, 31-Jul-2015, paragraph 2.1, page 28:Primary objective:The primary objective is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.Secondary objectives:* Evaluation of…

HOMERUN
Initiation of HOme MEchanical ventilation at home in a selective group of patients with chronic hypercapnic Respiratory failUre in the Netherlands

Patients with chronic respiratory failure currently start their ventilatory support in hospital as stated in national guideline. However, as these patients are severely disabled, a stay at the hospital for several days is experienced by many as very…

A randomized, double blind, double-dummy placebo controlled, 3-way cross-over study to determine the test-retest reliability of, and the effect of oral retigabine and riluzole on, peripheral motor nerve excitability measurements in patients with ALS.

Patients with ALS are routinely prescribed riluzole, the only registered drug for the treatment of ALS. This study will evaluate the concentration-effect relationship of riluzole (100 mg) and retigabine (300 mg) on axonal excitability measures. In…

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…

A Phase 3, multi-center, randomized, Double-Blind, double-dummy, active controlled, parallel group study to evaluate the efficacy and safety of RPC1063 administered orally to relapsing multiple sclerosis patients.

Primary Objective:To assess whether the clinical efficacy of RPC1063 is superior to interferon (IFN) *-1a (Avonex®) inreducing the rate of clinical relapses in patients with RMS.Secondary Objectives:- To assess the effect of RPC1063 on the…

The effect of tetanus revaccination in patients with myasthenia gravis

The main objective of this study is to investigate the effectiveness of the humeral and cellular immune response after tetanus revaccination in patients with AChR MG, MuSK MG, or LEMS. The secondary objective is to determine if revaccination induces…

Evaluation of hand function in patients with Duchenne muscular dystrophy, including an additional pilot study with a newly designed glove. An explorative study.

To examine the natural disease course in the hands of persons with DMD and to explore relations between the level of body function/structures and the level of activities, according to the ICF model.

MR imaging of the spinal cord in patients with spinal muscular atrophy (SMA) and healthy controls

To investigate the potential value of magnetic resonance imaging (MRI) and diffusion tensor imaging (DTI) on a 3 Tesla MRI system to visualize the spinal cord in SMA patients and to investigate motor connectivity in vivo in patients with SMA.

An open label, cross over pilot study to evaluate the effect of a single dose of TW001 on the oxidative stress and pharmacokinetics with and without food in patients with amyotrophic lateral sclerosis

The aim of this study is to evaluate the effect of a single oral dose of TW001 on oxidative stress and pharmacokinetics with and without food. Dosing with an oral solution is considered to have major advantages over iv dosing in a chronic condition…

Play with the IROMEC robot during rehabilitation and special education for children with severe physical disabilities - an explorative feasibility study.

This study aims to examine the feasibility and an indication of possible effects of an IROMEC robot based play therapy within rehabilitation and special education on the level of playfulness of children with severe physical disabilities.

Effects of ODM109 on respiratory function in patients with ALS. A
randomised, double blind, placebocontrolled, crossover, 3 period,
multicentre study with openlabel followup extension.

The primary objective of this study will investigate the effect of oral levosimendan on the respiratory function of patients with Amyotrophic Lateral Sclerosis.

A natural history study on infantile facioscapulohumeral muscular dystrophy

The primary objective is to describe the clinical characteristics of infantile FSHD. The secondary objectives are: - To determine the incidence and prevalence of infantile FSHD- To describe the genetic characteristics of infantile FSHD. - To…

Functional effects of botulinum toxin in the hip adductors and subsequent exercise in patients with hereditary spastic paraplegia

This study aims to provide evidence for the notion that reduction of spasticity by bilateral BTX injections in the hip adductor group followed by self-administered stretching exercises and agility training improves lateral stepping and gait…

Precision orthotics: optimizing ankle foot orthoses to improve gait in neuromuscular diseases

To determine whether AFOs optimized for stiffness in patients with calf muscle weakness are superior to standard AFOs in reducing walking effort, as measured by energy cost of walking (ECW, in Jkg-1m-1), and 2) to build a model to predict required…