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=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=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=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=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
N/A N=22 Regulation of Muscle Protein Phenotype in Humans With Obesity
Obesity
Primary: Whole-Muscle Protein Synthesis — 0.004; 0.015 %/hr
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
Phase 3 N=90 A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Pompe Disease (Late-onset) · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Summary of Patients Reporting Treatment-Emergent Adverse Events — 60; 30; 32; 17 participants
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=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
Phase 2 N=75 A Study In Adults With Moderate To Severe Dermatomyositis
Dermatomyositis
Primary: Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 12 (Stage 1, Stage 2 and Amended Stage 2) — -3.44…
Phase 3 N=230 Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Muscular Dystrophy, Duchenne · Muscular Dystrophies · Muscular Disorders, Atrophic
Primary: Change From Baseline in 6MWD at Week 48 — -60.67; -47.69 meters — p=0.213
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
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 2 N=201 A Study in Older Participants Who Have Fallen and Have Muscle Weakness
Muscle Weakness
Primary: Change From Baseline to 24 Week Endpoint in Appendicular Lean Body Mass (aLBM) — 0.303; -0.123 kilograms (kg) — p=<0.001
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
Phase 2 N=212 An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
Primary Mitochondrial Myopathy
Primary: Change in Distance Walked During a 12 Minute Walk Test — 26.75; 30.89 meters
Phase 2 N=19 A Trial of PF-06252616 in Ambulatory Participants With LGMD2I
LGMD2I
Primary: Incidence of Dose Limiting or Intolerability Treatment Related Adverse Events — 45; 21; 40; 0 events
Phase 3 N=93 Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG
Myasthenia Gravis, Generalized
Primary: Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results — 4.96; 3.86; 6.14…
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
Phase 3 N=10 Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis
Sporadic Inclusion Body Myositis (sIBM)
Primary: Number of Participants With Adverse Events as a Measure of Safety and Tolerability — 0; 2; 10 Participants
N/A N=37 Forearm Immobilization, Metabolic Health, and Muscle Loss
Healthy
Primary: Percent Change in Forearm Glucose Uptake — -73; 127; -95 Percent change in clamp FGU with immob
Phase 2 N=36 Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Mitochondrial Myopathy
Primary: Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) — 13.5; 36.5; 64.5; 20.4 meters
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=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
Phase 3 N=251 Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients
Sporadic Inclusion Body Myositis
Primary: Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52 — 8.63; 9.63; -10.27; -8.96 meters — p=0.2210
N/A N=14 Muscle Capillarization and Sarcopenia
Sarcopenia
Primary: Skeletal Muscle Capillarization — 0.4; 0.2 capillaries per muscle fiber — p=0.27
Phase 2 N=30 Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
Primary Mitochondrial Disease
Primary: Distance Walked on the 6-minute Walk Test (6MWT) by Visit — 394.1; 378.2 meters
Phase 1 N=20 Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
Congenital Muscular Dystrophy
Primary: Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil — 1.05; 4.75; 2.15; 3.27 ng/mL
Phase 2 N=174 Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Duchenne Muscular Dystrophy · Becker Muscular Dystrophy
Primary: Change From Baseline in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756