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An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.

A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease

The primary objective of this study is to compare respiratory muscle strength values obtained using volitional techniques with values obtained using non-volitional techniques. The secondary objective is to compare the change for these two sets of…

EMG registration of the pelvic floor and pelvic floor muscle activity patterns during, abdominal, hip adductor, and gluteal muscle contractions in healthy women

To describe the EMG activity of the pelvic floor muscles (PFM) with a bi-polar EMG probe and the MAPLe and effect on the registered EMG activity with the probes in relation to the contractions of the muscles surrounding the pelvic floor. To describe…

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy

The primary objective is to test the hypothesis that once daily tadalafil administered orally for 48 weeks lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD). Two…

A randomized, two-arm, prospective, subject blind study to assess the safety and efficacy of InSpace* device in comparison to full thickness massive rotator cuff repair in subjects scheduled for a repair surgery.

This is a post-marketing study to further assess the safety and efficacy of the InSpace* device implantation in comparison to surgical repair of full thickness MRCT . The efficacy will be assessed by comparing the pre- and post- operative shoulder…

Abduction brace versus sling after arthroscopic cuff repair

Primary Objective: Is there reduction in pain using an abduction brace in patients undergoing arthroscopic cuff repair? Secondary Objectives: - Are there differences in function and recovery of the cuff, determined using questionnaires and echo?- Is…

A Multi-Center, Single-Blind, Randomized Study Comparing Thymectomy to No Thymectomy in Non-Thymomatous Myasthenia Gravis (MG) Patients Receiving Prednisone

The purpose of this 5-year trial is to determine if the surgical procedure, extended transsternal thymectomy (ETTX), combined with prednisone therapy is more beneficial in treating individuals with non-thymomatous MG than prednisone therapy alone.

Vitamin D suppletion and aspecific musculo-skeletal disorders

To investigate the effect of suppletion of vitamin D on aspecific musculo-skeletal disorders

Five year single blind effectiveness randomised actively controlled Phase III Clinical Trial in New Onset Juvenile Dermatomyositis: Steroids Treatment versus Steroids plus Cyclosporine Treatment versus Steroids plus Methotrexate Treatment

Objectives. The goals of the current protocol is therefore the natural follow-up of the objectives achieved with the previous grants and, in particular, of projects designed to discern new models for the successful conduct of clinical trials in…

An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.

Treatment of Myogenic Cranio Mandibular Dysfunction (CMD):
a prospective randomised clinical trial, comparing a mechanical stretching device (TheraBite®) with standard physiotherapy

To evaluate if a mechanical stretch device is effective in treating myogenic disturbances of the jaw joints, as compared with fysiotherapy.

Preoperative high-intensity physical training for elderly patients who are scheduled for elective abdominal oncological surgery

The objective of this pilot study is to investigate the feasibility and effect of a high-intensity physical training program for elderly patients who are scheduled for abdominal oncological surgery on the preoperative maximal aerobic capacity and…

Extension of the CBYM338B2203 phase IIb/III study to evaluate the long-term efficacy, safety and tolerability of intravenous BYM338 in patients with sporadic inclusion body myositis

Main Objective:To evaluate the long-term safety and tolerability of BYM338 in the treatment of sIBM and to further evaluate the effect of three BYM338dose regimens against placebo in increasing the distance traveled as measured by the 6 Minute…

A Phase III Open Label Extension Study to Assess the Long-Term Safety
and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy
(DMD) who completed the SIDEROS study

Primary:* To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.Secondary:* To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study,…

A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

This study has been transitioned to CTIS with ID 2023-507146-91-00 check the CTIS register for the current data. Primary Objective:• To evaluate the safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg…

A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids

Primary:* To assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using…

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

Primary:• To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (…

Personalized muscle training and biomarkers in children and adolescents with neuromuscular disease

Primary Objective:1) To determine the efficacy of personalized progressive resistance training (PRT) on muscle strength of elbow flexors in children and adolescents with Spinal Muscular Atrophy and Congenital Myopathy compared to usual care…

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis.

This study has been transitioned to CTIS with ID 2023-508284-77-00 check the CTIS register for the current data. The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of gMG in adults with autoantibodies…

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

This study has been transitioned to CTIS with ID 2024-511852-42-00 check the CTIS register for the current data. Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or…