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A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

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…

A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

Primary objective:To assess the efficacy of BMN 045 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives:To assess the safety and tolerability of BMN 045 after 48 weeks of treatment in all study…

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophy

Primary objective: To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives: - To assess the safety and tolerability of BMN 053 after single…

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…

Arm training in boys with Duchenne Muscular Dystrophy

To determine whether 3D arm training with arm-support is effective in delaying the loss of arm functions in daily activities from an early age onwards in boys with DMD.

Assessment of asymmetrical innervation of the right and left m. puborectalis as a possible cause of idiopathic incontinence, by using concentric needle electrodes in an electromyography investigation of the pelvic floor muscles.

Is it possible to make a discrimination in muscle activity between the right and left puborectalis muscle by using concentric needle electrodes in an electromyography investigation of the pelvic floor muscles?When a asymmetry is found, what can be…

pharmacokinetic-pharmacodynamic modeling of S(+)-ketamine in fibromyalgia

To test the hypothesis that ketamine has a modulatory effect on fibromyalgia

Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy

Untill now, MRS has never been applied to skeletal muscles of FSHD patients. As MRS provides a unique window on several compounds in skeletal muscle and some skeletal muscles of FSH patients are affected by the disease, it is possible that…

A phase II, open-label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

The objective of the study is to assess the safety, tolerability and efficacy of PRO044.

Study the dose and effectiveness of dietary sugar supplementation in congenital muscular dystrophy

The primary objective is to determine the effect of dietary ribose supplementation on muscle function in patients with isolated LGMD as caused by a dystroglycanopathy. The secondary aim is to determine if ribose is well tolerated in this patient…

Assess effect and safety of intra-arterial autologous mesoangioblasts administration to the upper arm of m.3243A>G mutation carriers

This study has been transitioned to CTIS with ID 2024-515129-27-00 check the CTIS register for the current data. In our phase IIa trial we will determine the effect and safety of three MABs administrations in the m. biceps brachii in m.3243A>…

Feasibility of a 13-week resistance exercise and protein supplementation intervention in sarcopenic geriatric rehabilitation inpatients: EMPOWER-GR

The objective of the study is to investigate the feasibility of a combined resistance exercise training (RET) and protein supplementation (ONS) intervention and explore the effect on muscle mass and other parameters compared to standard of care in…

An Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with Extension

This study has been transitioned to CTIS with ID 2024-512736-29-00 check the CTIS register for the current data. Primary Objective1. To evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1Secondary…