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
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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
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
Phase 2 N=152 Study of Arimoclomol in Inclusion Body Myositis (IBM)
Inclusion Body Myositis
Primary: Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score — -3.26; -2.26 score on a scale — p=0.1146
Phase 3 N=211 An Extension Study of the Efficacy, Safety and Tolerability of BYM338 (Bimagrumab) in Patients With Sporadic Inclusion Body Myositis Who Previously Participated in the Core Study CBYM338B2203
Sporadic Inclusion Body Myositis
Primary: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths. — 48; 50; 44; 49 Participants
Phase 3 N=17 Belimumab in Idiopathic Inflammatory Myositis
Myositis
Primary: Response Rate During Randomized Phase — 3; 1; 5; 2 Participants — p=0.60
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 3 N=89 Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Hereditary Inclusion Body Myopathy · Distal Myopathy With Rimmed Vacuoles · Distal Myopathy, Nonaka Type
Primary: Change From Baseline in UEC Score (Total Force in kg) at Week 48 — -2.25; -2.99 kg — p=0.5387
Phase 2 N=9 Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Multiple System Atrophy
Primary: Number of Adverse Events up to Six Months Post-treatment — 43; 0 Adverse events
Phase 3 N=148 Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy
Polymyositis · Dermatomyositis · Autoimmune Necrotizing Myopathy
Primary: Number of Participants Achieving International Myositis Assessment and Clinical Studies Definition of Improvement (IMACS DOI) at Week 24 Without Rescue — 42; 31…
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 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 2 N=37 Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
Spinal and Bulbar Muscular Atrophy
Primary: Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability — 2; 4; 2; 1 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 2 N=50 Dutasteride to Treat Spinal and Bulbar Muscular Atrophy (SBMA)
Kennedy's Disease · Spinal and Bulbar Muscular Atrophy
Primary: Muscle Strength Change From Baseline — -2.2; 3.1; -4.5; 1.3 percent change — p=0.28
N/A N=89 Electrical Impedance Myography as an Outcome Measure in Amyotrophic Lateral Sclerosis Clinical Trials
Amyotrophic Lateral Sclerosis
Primary: Electrical Impedance Myography — 0.55 Coefficient of Variation
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
N/A N=53 Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Spinal Muscular Atrophy (SMA)
Primary: Motor Function Assessments- Test for Infant Motor Performance Screening Items (TIMPSI) — 36.06; 88.47; 40.54; 89.44 scores on a scale
Phase 2 N=217 Dose Range Finding Study of Bimagrumab in Sarcopenia
Sarcopenia
Primary: Change From Baseline in Total Short Physical Performance Battery (SPPB) Score to Week 25 — 7.1; 7.3; 7.2; 7.3 Score on a scale — p=0.274
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
N/A N=95 Identifying Therapeutic Targets of Accelerated Sarcopenia
Sarcopenia · Diabetes Mellitus · Aging
Primary: Leg Lean Mass — 0.35; -0.55; -0.80; -0.44 kg — p=<0.01
Phase 2 N=54 A Study of NIDO-361 in Patients With SBMA
Spinal and Bulbar Muscular Atrophy · Kennedy's Disease
Primary: Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] — 4.1402; 3.8580; -0.0871; -0.1042 cm³
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
N/A N=22 Regulation of Muscle Protein Phenotype in Humans With Obesity
Obesity
Primary: Whole-Muscle Protein Synthesis — 0.004; 0.015 %/hr
N/A N=25 Rehabilitation of Idiopathic Pulmonary Fibrosis (IPF) Patients
Idiopathic Pulmonary Fibrosis
Primary: 6 Minute Walk Distance — -6.2; -15.3 meters
Phase 2 N=20 Abatacept for the Treatment of Myositis-associated Interstitial Lung Disease
Myositis · Interstitial Lung Disease
Primary: % Predicted Forced Vital Capacity (FVC) Absolute Change — -1.70; -2.05 % predicted FVC — p=<0.05
Phase 2 N=142 Comparison of Therapeutic Regimens for Scleroderma Interstitial Lung Disease (The Scleroderma Lung Study II)
Scleroderma · Interstitial Lung Disease
Primary: Forced Vital Capacity (FVC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value — 66.52; 66.52; 66.22; 67.03 FVC %-pred — p=0.24
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
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=6 A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
Limb-Girdle Muscular Dystrophy, Type 2D
Primary: Number of Participants With Adverse Events (AEs). — 1; 3; 2; 0 Participants