Phase 3 N=24 Arimoclomol in Sporadic Inclusion Body Myositis
Inclusion Body Myositis
Primary: Count of Adverse Events Reported — 109; 52 adverse events reported
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=45 The Exercise Response to Pharmacologic Cholinergic Stimulation in Myalgic Encephalomyelitis / Chronic Fatigue Syndrome
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome · Chronic Fatigue Syndrome · Myalgic Encephalomyelitis
Primary: Change in Peak Oxygen Uptake (Peak VO2) Between the First and Second iCPET — 13.3; -40.3; 111.8 mL/min — p=0.043
N/A N=6 Duchenne Muscular Dystrophy Clinical Trial
Duchenne Muscular Dystrophy
Primary: Percent Change in Range of Motion From Baseline to 180 Days — 67; 33 percentage of change
Phase 2 N=63 Antioxidant Therapy in RYR1-Related Congenital Myopathy
Neuromuscular Disease
Primary: Urine 15-F2t Isoprostane Concentration — 2.7; 2.6 ng/mg Cr — p=0.88
Phase 2 N=59 Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia
Myotonia · Non-Dystrophic Myotonia
Primary: Patient-reported Stiffness on the IVR — 2.53; 4.21; 1.60; 5.27 units on a scale — p=<0.001
Phase 2 N=53 RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
MItochondrial Myopathies
Primary: Change of Peak Workload (in Watts/kg) During Exercise Testing — 0.0090; 0.020; -0.0300; 0.1050 Watts/kg — p=0.7321
Phase 2 N=62 A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis
Myasthenia Gravis, Generalized
Primary: Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score. — -4.6; -2.7 units on a scale
Phase 1 N=7 S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
RYR-1 Myopathy
Primary: Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) — 0; 0; 0; 0 Participants
Phase 4 N=10 Examining the Effect of Burosumab on Muscle Function
X-linked Hypophosphatemia
Primary: Skeletal Muscle Adenosine Triphosphate (ATP) Synthesis Rate — 9.41 umol/g/min — p=0.4147
Phase 4 N=16 Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD II) · Glycogenesis 2 Acid Maltase Deficiency
Primary: Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26 — 5.3; -1.6 percent area occupied by glycogen — p=0.1860
N/A N=27 Composition & Function of Sarcoplasmic Reticulum in Persons With the Metabolic Syndrome
Metabolic Syndrome
Primary: Sarcoplasmic Reticulum Composition — 2.0; 2.2 PC:PE ratio
N/A N=11 Electromyography in Patients on Chronic Pyridostigmine Therapy
Muscle Weakness
Primary: Percent Change in the Amplitude of Evoked Compound Muscle Action Potential by Electromyography Between the First to Fifth Response — 0 percent change
Phase 2 N=17 Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
Myotonic Dystrophy
Primary: The Number of Study Participants Who Safely Tolerated Somatokine — 6; 9 participants
Phase 2 N=4 Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Charcot Marie Tooth Disease
Primary: Number of Participants With a Decrease in Cramp Duration — 2; 0 participants
Early Phase 1 N=32 Premature Fatigue in Veterans With Heart Failure: Neuronal Influences
Heart Failure
Primary: Maximal Voluntary Quadriceps Force [% Change From Baseline] — -30; -5 percentage change
N/A N=8 7 Tesla MRI Study in Patients With Statin Related Muscle Complaints
Statin-related Muscle Pain · Muscle Aches · Muscle Weakness
Primary: Change in Pre- and Post-phosphocreatine Relaxation Time — 5.58; 5.083 milliseconds
Phase 2 N=69 Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1
Primary: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance — 12.40; 20.11 meters
Phase 3 N=39 Clinical Trial of CoQ10 for Mild-to-Moderate Statin-Associated Muscle Symptoms
Pain · Weakness · Cramps
Primary: Percentage of Participants With Reduction in Muscle Pain Associated With Statin Use — 53; 65 % of participants with pain reduction
Phase 2 N=42 Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
Myotonic Dystrophy
Primary: Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance — 17.44; 7.25 Meters
Phase 2 N=12 An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy
GNE Myopathy
Primary: Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted) — 7461; 9432 hr*ng/mL
Phase 2 N=30 Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
Myotonic Dystrophy 1 · Excessive Daytime Sleepiness
Primary: Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score — -3.8; -1.8; 0.0 score on a scale
N/A N=14 Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Pompe Disease
Primary: Inspiratory Load Compensation - Inspiratory Volume — 288.41; 554.34; 743.71; 927.57 mL
Phase 2 N=70 A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
Spinal Muscular Atrophy
Primary: Change From Baseline to Week 8 in Forced Vital Capacity (FVC) — -0.02; -0.03; -0.07 liters — p=0.9086
N/A N=37 Amyloidopathy, Cholinopathy, Dopamine Responsiveness and Freezing of Gait in PD
Parkinson's Disease
Primary: L-DOPA Insensitivity — 5 Participants — p=0.00468
N/A N=92 DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Duchenne Muscular Dystrophy
Primary: Number of Subjects With an Adverse Event. — 1; 0; 0; 0 participants
N/A N=26 Gastrointestinal Symptoms in Postural Orthostatic Tachycardia Syndrome
Postural Tachycardia Syndrome
Primary: C-peptide Levels After Oral Glucose Tolerance Test — 40.6; 23.2 pg/mL
N/A N=22 Diagnosing Intensive Care Unit (ICU) Acquired Weakness
ICU Acquired Weakness
Primary: Peak Acceleration Measured in Extensor Carpi Radialis Longus — 0.476; 1.639; 0.541; 1.608 units of Gravity
N/A N=99 Inflammatory Abnormalities in Muscle After Stroke: Effects of Exercise
Stroke · Inflammation
Primary: Cardiovascular Fitness (VO2 Peak) — 15.9; 16.6; 21.3; 17.5 ml/kg/min — p=0.001
Phase 1 N=12 Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Myotonic Dystrophy, Type 1 (DM1) · Myotonic Dystrophy
Primary: Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo — 0; 0; 1; 1…
Phase 2 N=20 Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy (FSHD)
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 3; 6; 6; 5 Participants