Phase 2 N=2 (-)- Epicatechin Becker Muscular Dystrophy
Becker Muscular Dystrophy
Primary: Plasma Follistatin
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=3 Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Primary: Number of Dose Limiting Toxicity (DLT) Adverse Events as Assessed by 21 CFR 312.32. — 0 Number of Events
Phase 2 N=3 AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Duchenne Muscular Dystrophy
Primary: Number of Participants With Unacceptable Toxicity. — 0 Participants
Phase 2 N=15 Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
Duchenne Muscular Dystrophy
Primary: Pharmacokinetics Outcome: Absolute Values of (+)-Epicatechin Serum Concentration, Pre-dose (Trough) and 2 Hours Post-dose (Peak) — 15.1; 33.9; 20.6; 481.2 nM — p=0.0482
Phase 3 N=31 Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy
ATTR-CM · TTR-CM
Primary: Percentage of Participants With Categories of Patient Global Assessment (PGA) for Follow-up Visit: Month 12 — 0; 16.1; 9.7; 51.6 percentage of participants
Phase 2 N=3 Dose Confirmation Trial of AAV5-hFIXco-Padua
Hemophilia B
Primary: Factor IX Activity Levels — 30.6 Factor IX activity (%)
Phase 2 N=5 Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
Recessive Dystrophic Epidermolysis Bullosa
Primary: Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. — 5; 5; 4; 4 Fluorescence Intensity (MFI) for C7
Phase 2 N=9 Phase 2a Evaluation of PLN-74809 on αvβ6 Receptor Occupancy Using PET Imaging in Participants With IPF/
Idiopathic Pulmonary Fibrosis
Primary: Number of Participants With a Predicted Effect on αVβ6 PET ( Positron Emission Tomography) in Lungs After Administration of Drug. — 1; 1; 0; 2 Participants
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
N/A N=117 Alpha-1 Carrier Genomics Study
Alpha-1 Antitrypsin Deficiency · Emphysema · COPD
Primary: Number of Participants With Abnormal Sequences in SERPINA1 Genes — 6; 0 participants — p=0.054
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=11 Gene Therapy for Achromatopsia (CNGA3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
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
N/A N=40 High-Purity Type I Collagen Nerve Wrapping in Peripheral Nerve Procedures: A Prospective Registry
Peripheral Nerve Injuries · Neuroma of Nerve Repair · Nerve Graft; Complications, Mechanical
Primary: Motor Functional Recovery (MRC Grade ≥ M3) — 34 Participants
Phase 3 N=25 A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
Symptomatic Transthyretin Amyloid Cardiomyopathy
Primary: Part A: Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT) — -3.86 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 2 N=13 Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Primary: Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 13; 5; 7; 7 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
Phase 2 N=6 Gentamicin for RDEB
Recessive Dystrophic Epidermolysis Bullosa
Primary: Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence — 31.8; 28.4; 65.6 C7 Expression (percent of normal skin)
Phase 2 N=21 The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis
Transthyretin-associated Amyloidosis With Polyneuropathy
Primary: Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer at Week 6 — 94.7 percentage of participants
Phase 2 N=23 Gene Therapy for Achromatopsia (CNGB3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Phase 2 N=7 Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica · Epidermolysis Bullosa
Primary: Number of Wounds by Healing Category Per Investigator Visual Assessment — 35; 5; 2; 28 wounds
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 4 N=347 Effect of Nintedanib on Biomarkers of Extracellular Matrix Turnover in Patients With Idiopathic Pulmonary Fibrosis and Limited Forced Vital Capacity Impairment
Idiopathic Pulmonary Fibrosis
Primary: The Rate of Change (Slope) in Blood C-reactive Protein Degraded by Matrix Metalloproteinase-1/8 (CRPM) From Baseline to Week 12. — -0.00190; -0.00257 nanogram/ millitre/…
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
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 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 2 N=13 A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)
C3 Glomerulopathy · C3 Glomerulonephritis · Dense Deposit Disease
Primary: Change From Baseline In Composite Biopsy Score At Week 28 — 11.7; 9.3; 9.2; 10.7 score on a scale
N/A N=18 High-Purity Type I Collagen Augmentation in Meniscal Repair
Meniscus Tear · Knee Meniscus Injury · Meniscal Repair
Primary: Safety of High-Purity Type I Collagen Augmentation — 0; 0; 0; 0 Participants
Phase 1 N=6 Eculizumab Therapy for Dense Deposit Disease and C3 Nephropathy
Dense Deposit Disease · Membranoproliferative Glomerulonephritis
Primary: Number of Patients With Change in Proteinuria or Serum Creatinine Over Treatment Period — 5 Participants