Clinical trials: MYOPATHY
Rheumatoid Arthritis, Myositis
Rheumatoid Arthritis and Myositis Cohort
The primary objective of this research is to establish a well characterized clinical and longitudinal cohort for individuals with Rheumatoid Arthritis (RA) and Myositis to create a place to maintain blood, urine, stool specimens…
- Trial name
- NCT05627089
- Sponsor
- Attune Health Research, Inc.
Healthy, Idiopathic Inflammatory Myopathies
A Phase 1 Open-label Study to Evaluate Safety in Healthy Participants and Participants With Autoimmune Diseases
The purpose of this study is to determine the safety and tolerability of XmAb657 in healthy participants and participants with autoimmune diseases.
- Conditions
- Healthy, Idiopathic Inflammatory Myopathies, Systemic Sclerosis, Sjögren's Disease
- Trial name
- NCT07284797
- Testing
- XmAb657
- Where
- Australia
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Xencor, Inc.
Systemic Lupus Erythematosus, Rheumatoid Arthritis
Early Clinical Study of UTAA91 Injection for the Treatment of Relapsed/Refractory Autoimmune Diseases
This clinical trial is designed as a single - arm, open - label, single - center, investigator - initiated early - phase clinical study.
- Conditions
- Systemic Lupus Erythematosus, Rheumatoid Arthritis, Dry Syndrome, Idiopathic Inflammatory Myopathies
- Trial name
- NCT06970951
- Testing
- UTAA91 injection
- Where
- China
- Volunteers
- 15 people
- Sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.
GNE Myopathy, Hereditary Inclusion Body Myopathy
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA).
- Trial name
- GNE Myopathy (NCT01517880)
- Testing
- Sialic Acid Extended Release (SA-ER)
- Where
- 4 sites in United States, Israel
- Volunteers
- 46 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies
Early Clinical Study of UTAA09 Injection in the Treatment of Relapsed/Refractory Autoimmune Diseases
Main purpose: To evaluate the safety of UTAA09 injection in the treatment of relapsed/refractory (R/R) autoimmune disease (AID).
- Conditions
- Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies, Systemic Sclerosis, IgG4 Related Disease
- Trial name
- NCT06361745
- Testing
- T cell injection targeting CD19 chimeric antigen receptor
- Where
- China
- Volunteers
- 10 people
- Sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Muscular Dystrophies
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
- Trial name
- NCT01480245
- Testing
- GSK2402968
- Where
- 59 sites in Argentina, Australia and 22 more
- Volunteers
- 233 people
- Sponsor
- GlaxoSmithKline
Inclusion Body Myositis
Study of Arimoclomol in Inclusion Body Myositis (IBM)
Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.
- Trial name
- NCT02753530
- Testing
- Arimoclomol
- Where
- 12 sites in United States, United Kingdom
- Volunteers
- 152 people
- Sponsor
- ZevraDenmark
Fructose Metabolism, Inborn Errors, Glycogen Storage Disease
Biomarker for Glycogen Storage Diseases (BioGlycogen)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Glycogen Storage Diseases from plasma. Testing for clinical robustness, specificity and long-term stability of the biomarker.
- Conditions
- Fructose Metabolism, Inborn Errors, Glycogen Storage Disease, Glycogen Storage Disease Type I, Glycogen Storage Disease Type II
- Trial name
- BioGlycogen (NCT02385162)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Systemic Lupus Erythematosus, Lupus Nephritis (LN)
An Exploratory Clinical Study of YTS109 Cell for R/R Autoimmune Diseases
This study evaluates the safety and efficacy of YTS109 cells in adults with relapsed/refractory autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), including LN and SLE-ITP, Sjogren's Syndrome, etc.
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis (LN), Sjogren's Syndrome, Inflammatory Myopathy
- Trial name
- NCT07236762
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 18 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD).
- Trial name
- NCT05154851
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Systemic Lupus Erythematosus, Systemic Sclerosis
Clinical Study on Targeted CD19CAR-T Therapy for Autoimmune Diseases
This is an open-label, investigator-initiated clinical trial (IIT) aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with active SLE, SSc, AAV, IIM, and pSS
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis, ANCA Associated Vasculitis, Idiopathic Inflammatory Myopathies
- Trial name
- NCT06549296
- Testing
- RD06-04 CART Cell Injection
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Nanjing Bioheng Biotech Co., Ltd.
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-45 (NCT07038824)
- Testing
- ENTR-601-45
- Where
- 15 sites in Belgium, Italy and 3 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Post-traumatic Stress Disorder, Persian Gulf Syndrome
Sentra AM® and Sentra PM® for Post-traumatic Stress Disorder (PTSD) and Gulf War Fibromyalgia (GWF)
An open-label clinical outcomes study to determine the safety and efficacy of Sentra AM® and Sentra PM® for post-traumatic stress disorder (PTSD) and Gulf War fibromyalgia (GWF).
- Trial name
- GWF001 (NCT01437891)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Stephanie Pavlik
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
A Phase 2b Study of Zagociguat in Patients With MELAS
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs.
- Trial name
- PRIZM (NCT06402123)
- Testing
- zagociguat 15mg, zagociguat 30mg
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 43 people
- Sponsor
- Tisento Therapeutics
Fibromyalgia
Investigation of AVACEN Thermal Exchange System for Fibromyalgia Pain
Hypothesis: Daily use of the AVACEN Thermal Exchange System for 4 weeks will show improved Fibromyalgia pain and functioning markers, as assessed by biomarkers and clinical and psychological assessment.
- Trial name
- AVACEN: TES (NCT01619579)
- Testing
- AVACEN Thermal Exchange System
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Avacen, Inc.
Duchenne Muscular Dystrophy
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle.
- Trial name
- NCT01610440
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Becker Muscular Dystrophy
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
Objectives: Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months. Secondary Objectives: To establish the macroscopic muscle effects of Givinostat versus placebo…
- Trial name
- NCT03238235
- Testing
- Givinostat
- Where
- 2 sites in Italy, Netherlands
- Volunteers
- 51 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.