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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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 1 N=20 Rimeporide in Patients With Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne
Primary: Number of Participants With Adverse Events — 2; 1; 5; 4 Participants
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=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=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 3 N=24 Arimoclomol in Sporadic Inclusion Body Myositis
Inclusion Body Myositis
Primary: Count of Adverse Events Reported — 109; 52 adverse events reported
N/A N=120 Treatment of Critical Illness Polyneuromyopathy
Acute Respiratory Failure
Primary: The Primary Outcome Variable for This Study Will be the Short Form of the Continuous Scale Physical Functional Performance Test (CS-PFP) Called the PFP-10 — 19.0; 20.9…
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
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=45 Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
X-Linked Retinitis Pigmentosa
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to…
N/A N=39 ABCA3 Gene and RDS in Late Preterm and Term Infants
Respiratory Distress Syndrome, Newborn
Primary: Mortality — 5; 3; 5 Participants
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=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 2 N=12 Phase I/II Study of SRP-4053 in DMD Patients
Duchenne Muscular Dystrophy
Primary: Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation — 4; 5; 5; 3 Participants
Phase 2 N=36 Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Inherited Mitochondrial Disease, Including Leigh Syndrome
Primary: Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV — -0.3; 0.1; -0.6; 0.0 units on a scale — p=0.1875
Phase 1 N=12 Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 4; 5; 3; 3 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=8 Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral Muscular Dystrophy · Limb Girdle Muscular Dystrophy
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 7; 0 Participants
N/A N=9 Respiratory Muscle Training (RMT) in ALS
ALS
Primary: Number of Participants With Safe, Well-tolerated Moderate Intensity Respiratory Muscle Training (RMT) as Measured by Rating of Perceived Exertion (RPE) — 4 Participants
Phase 4 N=14 Ranolazine for the Treatment of Chest Pain in HCM Patients
Hypertrophic Cardiomyopathy
Primary: QT Interval — 462.8 msec
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=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 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=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 2 N=9 Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
Facioscapulohumeral Muscular Dystrophy
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 9; 0 Participants
Phase 2 N=17 A Study of RVT-1401 in Myasthenia Gravis (MG) Patients
Myasthenia Gravis
Primary: Double-Blind Treatment Period: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 5; 4; 5; 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=45 Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Primary: Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score — -5.5; -6.0; -3.2 score on a scale — p==0.0941
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…