Clinical trials: Myopathy
SLE (Systemic Lupus), Lupus Erythematosus, Systemic
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus…
- Conditions
- SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis, Systemic Sclerosis
- Trial name
- NCT06925542
- Testing
- CTX112
- Where
- 17 sites in United States, Germany and 1 more
- Volunteers
- 80 people
- Sponsor
- CRISPR Therapeutics
Systemic Lupus Erythematosus, Systemic Sclerosis
An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease
An exploratory clinical study of the safety and efficacy of YTS109 cell injection in subjects with recurrent/refractory autoimmune disease
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis, Inflammatory Myopathy, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Trial name
- NCT06379646
- Testing
- YTS109
- Where
- China
- Volunteers
- 6 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Autosomal Dominant RYR1-Related Myopathy
A Trial to Compare Treatment With Surlorian (ARM210, S48168) to Placebo in Effects on Muscle Strength and Safety in Adults With Autosomal Dominant RYR1-related Myopathy
This study is testing a medicine called surlorian in adults who have a genetic muscle condition known as autosomal dominant RYR1-related myopathy (RYR1-RM).
- Trial name
- NCT07560020
- Testing
- Surlorian
- Where
- 9 sites in France, Germany and 3 more
- Volunteers
- 28 people
- Sponsor
- RyCarma Therapeutics, Inc.
Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc)
A Clinical Study of YTS109 Cell in 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), Systemic Sclerosis (SSc), etc.
- Conditions
- Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), Inflammatory Myopathy, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Trial name
- NCT06978647
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 12 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Systemic Lupus Erythematosus (SLE), Lupus Nephritis (LN)
A 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 (SLE), Lupus Nephritis (LN), Systemic Sclerosis (SSc), Inflammatory Myopathy
- Trial name
- NCT07104721
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 18 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Systemic Lupus Erythematosus (SLE), Lupus Nephritis (LN)
A Clinical Study of YTS109 Cells for the Treatment of 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 (SLE), Lupus Nephritis (LN), Sjogren's Syndrome (SS), Inflammatory Myopathy
- Trial name
- NCT07123519
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 18 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Muscular Dystrophies, Muscle Myopathies
Genetic and Physical Study of Childhood Nerve and Muscle Disorders
Background: Some nerve and muscle disorders that start early in life (before age 25), like some forms of muscular dystrophy, can run in families. However, the genetic causes of these disorders are not known.
- Conditions
- Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies
- Trial name
- NCT01568658
- Where
- United States
- Volunteers
- 2,586 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies
A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 in Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies or Rheumatoid Arthritis.
The purpose of this study is to measure the safety, tolerability, PK, and PD of AZD5492 administered subcutaneously in adult participants with SLE or IIM or RA Study details include: The study duration will be a minimum of 180…
- Conditions
- Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies, Rheumatoid Arthritis
- Trial name
- TITAN (NCT06916806)
- Testing
- AZD5492
- Where
- 38 sites in United States, Canada and 8 more
- Volunteers
- 72 people
- Sponsor
- AstraZeneca
Systemic Lupus Erythematosus (SLE), Sjogren's Syndrome (SS)
A Clinical Study of TI-0032-III Injection in Patients With Relapsed and Refractory Autoimmune Diseases
This is an open-label, dose escalation study in patients with relapsed and refractory autoimmune diseases. Study drug, TI-0032-III injection, is composed of lipid nanoparticles (LNPs) targeting T cells that encapsulate circular…
- Conditions
- Systemic Lupus Erythematosus (SLE), Sjogren's Syndrome (SS), Systemic Sclerosis (SSc), Inflammatory Myopathy
- Trial name
- in vivo CAR-T (NCT07413341)
- Testing
- TI-0032-III injection
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Therorna
Myositis, Active Idiopathic Inflammatory Myopathy
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
The main purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with Idiopathic Inflammatory Myopathy (IIM) who previously participated in ARGX-113-2007.
- Conditions
- Myositis, Active Idiopathic Inflammatory Myopathy, Dermatomyositis, Polymyositis
- Trial name
- ALKIVIA+ (NCT05979441)
- Testing
- Efgartigimod PH20 SC
- Where
- 92 sites in United States, Argentina and 27 more
- Volunteers
- 240 people
- Sponsor
- argenx
Chronic Inflammatory Demyelinating Polyradiculoneuropathy, NMO Spectrum Disorder
Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases
This study is a phase Ib/IIa dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of autologous T cells expressing chimeric antigen receptor (CAR)-targeted B-cell activating factor…
- Conditions
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy, NMO Spectrum Disorder, Myasthenia Gravis, Idiopathic Inflammatory Myopathies
- Trial name
- NCT07022197
- Testing
- BAFF-R CART
- Where
- China
- Volunteers
- 27 people
- Sponsor
- Tianjin Medical University General Hospital
Idiopathic Inflammatory Myopathy, Dermatomyositis
RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
- Conditions
- Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy
- Trial name
- NCT06154252
- Testing
- CABA-201 following preconditioning with fludarabine and cyclophosphamide
- Where
- 38 sites in United States, Germany and 2 more
- Volunteers
- 74 people
- Sponsor
- Cabaletta Bio
Ryanodine Receptor 1-Related Myopathy, Ryanodine Receptor 1 Related Disorders
A Natural History Study of RYR1-Related Disorders
Background: Congenital myopathies (CM) are genetic disorders that can cause decreased muscle tone and muscle weakness. Most CMs in the United States are related to the ryanodine receptor 1 (RYR1) gene.
- Trial name
- NCT06287762
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Relapsed/Refractory Systemic Lupus Erythematosus, Relapsed/Refractory Systemic Sclerosis
Autologous CD19/BCMA Dual-Target CAR-T for Relapsed/Refractory Autoimmune Diseases
Autoimmune diseases occur when the immune system mistakenly attacks the body's own tissues, leading to chronic inflammation and damage to organs such as the kidneys, lungs, muscles, nerves, or blood cells.
- Conditions
- Relapsed/Refractory Systemic Lupus Erythematosus, Relapsed/Refractory Systemic Sclerosis, Relapsed/Refractory Idiopathic Inflammatory Myopathies, Relapsed/Refractory sjögren's Syndrome
- Trial name
- NCT07361094
- Testing
- Autologous CD19-BCMA Dual-Target CAR T-Cell Therapy
- Where
- China
- Sponsor
- Beijing Boren Hospital
X-linked Myotubular Myopathy
A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07876804
- Testing
- ASP2957
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Idiopathic Inflammatory Myopathies
A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies
This study is open to adults aged 18 and older who have a condition known as active idiopathic inflammatory myopathy (IIM) or myositis. People can participate if they have a specific type of myositis.
- Trial name
- VERANDA™-IIM (NCT07789015)
- Testing
- Nerandomilast
- Where
- 150 sites in United States, Argentina and 22 more
- Volunteers
- 270 people
- Sponsor
- Boehringer Ingelheim
Systemic Lupus Erythematosus, Sjögren's Disease
A Study of BMS-986553 in Participants With Refractory Autoimmune Diseases
The purpose of this study is to evaluate the safety and tolerability of BMS-986553, CD19 in vivo Chimeric Antigen Receptor (CAR) T cells, in participants with refractory autoimmune diseases including systemic lupus erythematosus…
- Conditions
- Systemic Lupus Erythematosus, Sjögren's Disease, Rheumatoid Arthritis, Idiopathic Inflammatory Myopathies
- Trial name
- NCT07873931
- Testing
- BMS-986553
- Where
- 23 sites in United States, Belgium and 4 more
- Volunteers
- 60 people
- Sponsor
- Bristol-Myers Squibb
Rheumatoid Arthritis, Systemic Lupus Erythematosus
Study of Families With Twins or Siblings Discordant for Rheumatic Disorders
This study will examine families in which one sibling of a sibling pair, or twin pair, has developed a systemic rheumatic disease and one has not, to see if and how the two differ in the following: Blood cell metabolism; Types of…
- Conditions
- Rheumatoid Arthritis, Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies
- Trial name
- NCT00055055
- Where
- 3 sites in United States
- Volunteers
- 1,056 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
GNE Myopathy
First-in-human Study of UX016 in GNEM
The goal of this study is to assess the safety and dose of UX016 and its impact on muscle strength in adults with GNE Myopathy (GNEM).
- Trial name
- NCT07511556
- Testing
- UX016
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
MItochondrial Myopathies, Mitochondrial Disease
Validation of Nanosensor Oxygen Measurement
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM).
- Trial name
- NCT06819683
- Testing
- Nanosensor
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia