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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…

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 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…

A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN PATIENTS WITH SPINAL MUSCULAR ATROPHY

This study has been transitioned to CTIS with ID 2023-506761-65-00 check the CTIS register for the current data. The purpose of this Phase II/III study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of…

The efficacy of botulinum toxin A injection in pelvic floor muscles in chronic pelvic pain patients: a double-blinded randomised controlled trial

The objective of this study is to investigate whether BTA injection in the pelvic floor muscle is an effective treatment for patients with chronic pelvic pain and pelvic floor hypertonicity.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy EPIDYS (Epigenetic Rescue of Dystrophin Dysfunction)

Primary objective:* To establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.Secondary objectives:* To assess the safety and tolerability of givinostat versus…

PROSPECTIVE, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED PHASE III STUDY EVALUATING EFFICACY AND SAFETY OF OCTAGAM 10% IN PATIENTS WITH DERMATOMYOSITIS

Primary Objective:The primary objective of this study is to provide confirmatory data on the beneficial effect of 2.0 g/kg of Octagam 10% given every 4 weeks compared with placebo in subjects with active DM based on the percentage of responders at…