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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 randomized, double-blind, placebo-controlled, multicenter, parallel group, dose-finding, pivotal, phase IIb/III study to evaluate the efficacy, safety and tolerability of intravenous BYM338 at 52 weeks on physical function, muscle strength, and mobility and additional long-term safety up to 2 years in patients with sporadic inclusion body myositis (CBYM338B2203)

Primary: to demonstrate that at least one dose regimen of BYM338 in sporadic inclusion body myositis patients will increase the distance traveled as measured by change from baseline at Week 52 of the 6 minute walking distance test relative to…

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…

A randomized, double blind, placebo controlled, multi-centre study to assess the pharmacodynamics, pharmacokinetics, safety and tolerability of BYM338 in chronic obstructive pulmonary disease patients with cachexia (CBYM338X2204)

Primary: Assess the effect of i.v. infusions of BYM338 on muscle volume of the thigh (assessed by MRI) at 4, 8, 16 and 24 weeks, compared to placebo, in COPD patients with pulmonary cachexia.Secondary: Effect on 6-minute walk test, safety and…

A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY OF THE SAFETY AND EFFICACY OF 3-MONTH SUBCUTANEOUS REGN1033 TREATMENT IN PATIENTS WITH SARCOPENIA

Please refer to protocol, section 1.2 "Rationale"

A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging, Dose-Loading Study to Evaluate the Efficacy, Safety, and Tolerability of JNJ-42160443 as Adjunctive Therapy in Subjects With Inadequately Controlled, Moderate to Severe, Chronic Low Back Pain

Primary ObjectivesThe primary objectives of this study are to evaluate the analgesic effect size over 12 weeks of several doses and dosage regimens of JNJ-42160443 compared with placebo in subjects with moderate to severe, chronic, low back pain (…

The impact of the macronutrient composition of a nutritional supplement on postprandial skeletal muscle protein synthesis in elderly

Obtain more insight in the preferred macronutrient composition of an oral nutritional supplement.

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…

Effects of levosimendan on diaphragm function in mechanically ventilated patients

To determine the effect of levosimendan on diaphragm function in mechanically ventilated patients.

Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia.

(voor meer details zie CMO-protocol paragraaf 2: Objectives) Primary Objective: The objective of this project is to further refine N-of-1 trial methodology, and explore whether Bayesian analysis of multiple N-of-1 trials can serve as a sufficient…

The effect of treatment of craniomandibular dysfunctions(CMD) in patients with long term neck complains;
A single blind randomized, controlled multicentral trial.

Recording if treatment of the craniomandibular region influences the sample of patients with long term neck complaints.

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…

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…

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…