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=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=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 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=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
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=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=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=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=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
N/A N=15 Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
Hereditary Spastic Paraplegia · Adrenomyeloneuropathy
Primary: Change From Baseline Walking Time in 10 Meter Walk Test to the Measurement Taken Directly After rTMS — -5.41; 1.70 seconds
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 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 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 3 N=22 Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
SMA - Spinal Muscular Atrophy · Gene Therapy
Primary: Achievement of Independent Sitting for at Least 30 Seconds — 13 Participants — p=<0.0001
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
Phase 2 N=18 L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Phase 2 N=2 (-)- Epicatechin Becker Muscular Dystrophy
Becker Muscular Dystrophy
Primary: Plasma Follistatin
Phase 2 N=12 Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
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=51 Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
Becker Muscular Dystrophy
Primary: Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies — -0.017; -0.054 log[percentage of total fibrosis]…
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 2 N=25 HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Duchenne Muscular Dystrophy · Cardiomyopathy
Primary: Number of Participants Experiencing Any of the Adjudicated Events — 0; 0; 0; 0 Participants
Phase 3 N=12 Betaine and Peroxisome Biogenesis Disorders
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Phase 2 N=35 Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Albinism · Inborn Errors of Metabolism · Oculocutaneous Albinism
Primary: Change in Forced Vital Capacity (36 Months) — -23.52; -20.93 % of predicted volume
Phase 2 N=109 Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
Huntington Disease
Primary: Safety and Tolerability of PBT2 in Patients With HD — 32; 30; 28 participants
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
Phase 2 N=7 Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
Becker Muscular Dystrophy
Primary: Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks — 0.55; 0.86 AU — p=0.0434
Phase 2 N=21 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615