Phase 3 N=53 Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Congenital Myotonic Dystrophy
Primary: Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) — -1.65; -3.40 score on a scale — p=0.0514
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=16 Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
Myotonic Dystrophy 1
Primary: Safety (Adverse Events) — 8; 6; 0; 0 Participants
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=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=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=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
Phase 2 N=41 Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Cardiomyopathy · Cardiomyopathy, Dilated
Primary: Incidence of Treatment-Emergent Adverse Events — 14; 9; 10 Participants
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
Phase 3 N=94 Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne Muscular Dystrophy · Becker Muscular Dystrophy · Dystrophinopathy
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 91; 31; 26 Participants
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=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
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…
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=312 A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Age of Participants When They Failed to Walk — NA; 14.4; 14.4; 11.1 Years
Phase 2 N=7 Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Number of Participants With Adverse Events Related to AVI-4568 — 0; 0 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
Phase 3 N=20 Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
Myasthenic Syndromes, Congenital
Primary: Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis — -0.13; -1.75; -0.75; -1.14 Scores on a scale…
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=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 3 N=360 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne · Muscular Dystrophies · Muscular Disorders, Atrophic
Primary: DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population — -81.83; -90.09 meters — p== 0.3626
Phase 2 N=25 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month) — 4.8 units on a scale
N/A N=42 Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: 12-month Change in Myocardial Strain — 2.2; 1.0 percent change in heart dimension
Phase 2 N=20 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Duchenne Muscular Dystrophy
Primary: Percent Change From Baseline in Dystrophin Level at Week 40, as Measured by ECL — 6.559 percent change
Phase 2 N=14 Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) — 14; 5; 8; 1 Participants
N/A N=53 Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Time to Sedation Score of 3-4 — 3.7; 2.8 minutes
Phase 1 N=16 Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
Muscular Dystrophies · Duchenne Muscular Dystrophy · Becker Muscular Dystrophy
Primary: Muscle, Nerve, or Vascular Damage — 16 participants
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…
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
N/A N=442 Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Phase 2 N=18 Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Primary: Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts — 1; 1…