Clinical trials are the final step in a long process of research. They allow scientists to test the value of their research to diagnose, treat and prevent diseases and disabilities. Each clinical trial is an opportunity to test a promising new invention or treatment in humans for the first time.

Currently recruiting

Cross-Sectional ALS Biofluid Biomarker (CABB) Study

Investigating Pompe Prevalence in NEuromuscular Medicine Academic Practices (IPANEMA Study)

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE 083 in Patients with Charcot-Marie-Tooth Disease Types 1 and X

Prospective, double-blind, randomized, placebo-controlled phase III study evaluating efficacy and safety of octagam 10% in patients with dermatomyositis (ProDERM study)

Expanded access protocol of patisiran for patients with hereditary transthyretin-mediated amyloidosis (HATTR amyloidosis) with polyneuropathy

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia

A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late-onset Pompe disease

Evaluation of the safety and efficacy of AMX0035, a fixed combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for treatment of Amyotrophic Lateral Sclerosis (ALS): A double blind placebo controlled Phase II study

Not yet recruiting

A phase 2, multi-center, double-blind, randomized, dose-ranging, placebo-controlled study to evaluate the efficacy, safety, and tolerability of CK-2127107 in patients with Amyotrophic Lateral Sclerosis (ALS)

Using Next Generation Sequencing to Unravel the Pathogenesis of Sporadic Inclusion Body Myositis (IBM) –The International IBM Consortium Genetic Study

A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects with Motor Neuron Disease

A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects with Charcot-Marie-Tooth Disease

Pompe disease registry protocol

Amyotrophic Lateral Sclerosis Association;Fulton Family Foundation

Genomic Translation for ALS Clinical care

Completed

Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS)

A phase III, open-label, extension trial of ECU-MG-301 to evaluate the safety and efficacy of eculizumab in subjects with refractory generalized Myasthenia Gravis (gMG)

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)

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

Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis

A randomized, double-blind, placebo-controlled, multi-center study to evaluate the safety and efficacy of eculizumab in subjects with refractory generalized Myasthenia Gravis (gMG)

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