Clinical trials: Myopathies
Sarcopenia, Critical Illness
Prevalence of Sarcopenia in Patients Admitted to ICU and Its Progression During Hospitalization.
This is a prospective, observational study at the ICU of Hospital Mútua de Terrassa, running from May to December 2025. The primary goal is to determine the prevalence of sarcopenia in adult patients requiring more than 48 hours…
- Conditions
- Sarcopenia, Critical Illness, Intensive Care (ICU) Myopathy, Body Composition Measurement
- Trial name
- SARC-ICU (NCT07303335)
- Where
- Spain
- Volunteers
- 100 people
- Sponsor
- Hospital Mutua de Terrassa
Systemic Lupus Erythematosus, Idiopahic Inflammatory Myopathies
A Study of CD19 UCAR-T Cells in Subjects With Autoimmune Diseases
The purpose of this study is to assess the safety and efficacy of CD19 UCAR-T cell therapy in Subjects with autoimmune diseases.
- Conditions
- Systemic Lupus Erythematosus, Idiopahic Inflammatory Myopathies, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Sjögren's Syndrome
- Trial name
- ET-902-AID01 (NCT07212322)
- Testing
- CD19 UCAR-T cells
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Metabolic Myopathies
Assessment of Energy Metabolism in Metabolic Myopathies
Patients with metabolic myopathies suffer from exercise intolerance due to impaired ability to produce energy and secondary de-conditioning.
- Trial name
- NCT07268287
- Testing
- 12-week Physiotherapy Intervention, 13C-Glucose Breath Test, Doubly labelled water & urine test
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- University of British Columbia
Immune-Related Adverse Events, Lupus Erythematosus, Systemic
Interferon Signature in Anti-CTLA-4 and Anti-PD-1/PD-L1-Treated Cancer Patients Compared With Systemic Autoimmune Disease Patients
This study aims to identify a way to predict the side effects that some people with cancer experience when receiving immunotherapy.
- Conditions
- Immune-Related Adverse Events, Lupus Erythematosus, Systemic, Sjogren Syndrome, Systemic Sclerosis (SSc)
- Trial name
- INTER-AUTENTIC (NCT07249060)
- Testing
- Immune Checkpoint Inhibitors
- Where
- Spain
- Volunteers
- 300 people
- Sponsor
- Hospital Universitario Araba
Mitochondrial Myopathies, Mitochondrial Diseases
Applying pGz in Mitochondrial Disease
This is a multi-aim study, studying the effects of conventional exercise (measured through Cardiopulomary Exercises Testing or an in-bed pedal exercise) and passive exercise through periodic acceleration (pGz).
- Trial name
- NCT05569122
- Testing
- Cardiopulmonary Exercise Testing, pGz Bed, Gentle Jogger
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Mitochondrial Myopathies, Mitochondrial Pathology
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases
- Trial name
- NuPower (NCT05162768)
- Testing
- Elamipretide
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 102 people
- Sponsor
- Stealth BioTherapeutics Inc.
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue.
- Conditions
- Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1
- Trial name
- NCT00082108
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Systemic Lupus Erythematosus, Systemic Sclerosis (SSc)
CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapse/Refractory Autoimmune Diseases
CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapse/Refractory Autoimmune Diseases
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis (SSc), Inflammatory Myopathy, ANCA-Associated Vasculitis (AAV)
- Trial name
- NCT06941129
- Testing
- UCAR T-cell
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Systemic Lupus Erythematosus (SLE), Idiopathic Inflammatory Myopathy (IIM)
Safety Study of CC312 in Autoimmune Disease Patients
This study is an open-label, multiple-dose escalation, Investigator-Initiated Trial (IIT) clinical trial designed to evaluate the safety and tolerability of CC312 in adult patients with relapsed and refractory autoimmune…
- Conditions
- Systemic Lupus Erythematosus (SLE), Idiopathic Inflammatory Myopathy (IIM), Systemic Sclerosis (SSc), Rheumatoid Arthritis (RA)
- Trial name
- NCT06888960
- Testing
- CC312
- Where
- China
- Volunteers
- 18 people
- Sponsor
- CytoCares Inc
Primary Mitochondrial Myopathies
Natural History in Primary Mitochondrial Myopathies
This is a longitudinal study in a cohort of patients with a genetic diagnosis of Primary Mitochondrial Myopathy to describe the natural history of the disease and identify clinical, biochemical, molecular, and radiological…
- Trial name
- NHPMM (NCT05653544)
- Where
- Spain
- Volunteers
- 150 people
- Sponsor
- Cristina Domínguez González
Idiopathic Inflammatory Myopathies(IIM), Immune Thrombocytopenia(ITP)
An Exploratory Study of CD19/CD22/BCMA CAR-T Cells (BZE2204) in Subjects With Relapsed or Refractory Autoimmune Diseases
This is a single arm, open-label, dose escalation and expansion study to evaluate the safety, tolerability and preliminary efficacy of autologous chimeric antigen receptor T (CAR-T) cells targeting CD19/CD22/BCMA(BZE2204) in…
- Conditions
- Idiopathic Inflammatory Myopathies(IIM), Immune Thrombocytopenia(ITP), Systemic Lupus Erythematosus(SLE)
- Trial name
- NCT07174843
- Testing
- CD19/CD22/BCMA CAR-T cells(BZE2204)
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shanghai Cell Therapy Group Co.,Ltd
Interstitial Lung Disease, Myopathy, Inflammatory
Tolerability and Safety of Nintedanib in Myositis Associated Interstitial Lung Disease
There is likely a role for using anti-fibrotic medications in patients with myositis-associated interstitial lung disease (MA-ILD) to slow down disease progression, especially in patients who have fibrotic and progressive…
- Trial name
- NCT05335278
- Testing
- Nintedanib 150 milligrams [Ofev]
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Rheumatic Diseases, Rheumatoid Arthritis (RA)
Clinical Assessment for Rheumatologic Disease - Research and Advancement in Safety and Efficacy
The CARe RAiSE project represents a pioneering translational initiative aimed at advancing precision medicine in the treatment of autoimmune rheumatic diseases.
- Conditions
- Rheumatic Diseases, Rheumatoid Arthritis (RA), Giant Cell Arteritis (GCA), Psoriatic Arthritis (PsA)
- Trial name
- CARe RAiSE (NCT07150000)
- Testing
- Ex Vivo Assay
- Where
- Germany
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Bonn
Duchenne or Severe Becker Myopathy
IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère
Clinical, prospective and monocentric study aiming at assessing the feasibility of fibrosis detection and quantification (and of function assessment) during MRI without breath-holds in a population of adults and children with…
- Trial name
- NCT02078076
- Testing
- Magnetic Resonance Cardiac Imaging (with Gadolinium)
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Phenotype, Genotype
Phenotype - Genotype Correlation in a Sample of Egyptian Patients With Congenital Myopathies and Congenital Muscular Dystrophies
The aim of this study is to correlate the phenotype and genotype among a sample of Egyptian patients with Congenital myopathies and Congenital muscular dystrophies.
- Conditions
- Phenotype, Genotype, Correction, Sample
- Trial name
- NCT07138963
- Testing
- Genetic Testing and Muscle Biopsy
- Where
- Egypt
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Ain Shams University
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Tobacco Use Disorder/Cigarette Smoking
Smoking Cessation Intervention for Young Adults Using MTM: A Randomized Controlled Trial
The goal of this clinical trial is to evaluate whether a Multi-Theory Model (MTM)-based behavioral intervention can effectively promote smoking cessation and support behavior change in young adult cigarette smokers aged 18 to 29…
- Trial name
- NCT07104006
- Testing
- Multi-Theory Model (MTM)-Based Smoking Cessation Program
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- University of Nevada, Las Vegas
Systemic Lupus Erythematosus (SLE), Immune-mediated Necrotizing Myopathy (IMNM)
A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy
This is an investigator-initiated, multicenter, open-label study of C-CAR168, an autologous bi-specific CAR-T therapy targeting CD20 and BCMA, for the treatment of adult patients with autoimmune diseases refractory to standard…
- Conditions
- Systemic Lupus Erythematosus (SLE), Immune-mediated Necrotizing Myopathy (IMNM), Neuromyelitis Optica Spectrum Disorders (NMOSD), Multiple Sclerosis (MS)
- Trial name
- CAR-AID (NCT06249438)
- Testing
- CD20/BCMA-directed CAR-T cells
- Where
- China
- Volunteers
- 30 people
- Sponsor
- RenJi Hospital
Systemic Sclerosis (SSc), Idiopathic Inflammatory Myopathy (IIM)
GC012F in Patients With Autoimmune Diseases
This is an open-label, early exploratory main clinical study to evaluate the safety and efficacy of GC012F Injection in subjects with autoimmune diseases (AID), as well as to assess its pharmacokinetic (PK) and pharmacodynamic…
- Trial name
- NCT07072884
- Testing
- GC012F injection
- Volunteers
- 15 people
- Sponsor
- Qiong Fu
Primary Mitochondrial Myopathy
A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy
The purpose of this study was to evaluate the dose response of Bocidelpar on functional improvement relative to placebo, safety, and tolerability in participants with Primary Mitochondrial Myopathy.
- Trial name
- MOUNTAINSIDE (NCT04641962)
- Testing
- Bocidelpar
- Where
- 11 sites in United States
- Volunteers
- 34 people
- Sponsor
- Astellas Pharma Inc