Clinical trials: Myopathies
Sjogrens Disease, Idiopathic Inflammatory Myopathy (IIM)
OM336 in Seropositive Autoimmune Diseases
An early-phase clinical trial evaluating the safety, tolerability, and pharmacokinetics of OM336 in adult participants with seropositive autoimmune diseases. OM336 is administered subcutaneously in ascending dose cohorts.
- Trial name
- NCT07229144
- Testing
- OM336
- Where
- New Zealand
- Volunteers
- 39 people
- Sponsor
- Lakefront Biotherapeutics West LLC
Inflammatory Rheumatism, Polymyalgia Rheumatica
PRediction Of DIverse Glucocorticoids toxIcity OUtcomeS
To date, there is no available tool that allows, at individual level, determination of the probability to develop clinically relevant complications of prolonged glucocorticoid therapy.
- Conditions
- Inflammatory Rheumatism, Polymyalgia Rheumatica, Giant Cell Arteritis, Idiopathic Inflammatory Myopathies
- Trial name
- PRODIGIOUS (NCT04664465)
- Where
- 12 sites in France
- Volunteers
- 300 people
- Sponsor
- University Hospital, Brest
Refractory/Relapsed Systemic Lupus Erythematosus, Refractory / Relapsed / Progressive Systemic Sclerosis
Allogeneic CD19/BCMA-Targeted CAR-γδT Cell Therapy: Safety and Preliminary Pharmacodynamics in Relapsed/Refractory Autoimmune Diseases
This study is a single-arm, intervention, dose-escalation clinical trial to evaluate the safety of allogeneic CD19/BCMA-targeted CAR-γδT cell in the treatment of relapsed/refractory autoimmune diseases
- Conditions
- Refractory/Relapsed Systemic Lupus Erythematosus, Refractory / Relapsed / Progressive Systemic Sclerosis, Refractory / Relapsing / Progressive Inflammatory Myopathy, Refractory / Relapsed Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis
- Trial name
- NCT07490275
- Testing
- Allogeneic CD19/BCMA-targeted CAR-γδT cell injection, Cyclophosphamide, Fludarabine
- Volunteers
- 9 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Myocarditis, ANCA Associated Vasculitis
EACVI Study on Multimodality Cardiovascular Imaging of Inflammatory Cardiovascular Diseases
Inflammatory Cardiovascular Diseases and Autoimmune Rheumatic Diseases (ICARDs) encompass cardiovascular involvement in connective tissue diseases, vasculitis, and primary inflammatory cardiac processes affecting all layers of…
- Conditions
- Myocarditis, ANCA Associated Vasculitis, Pericarditis, Systemic Sclerosis
- Trial name
- EACVI-INFLAME (NCT07077304)
- Testing
- observational study
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Systemic Sclerosis (SSc), Rheumatoid Arthritis (RA)
A Study of BEN301 Injection in theTreatment of Autoimmune Diseases
The study aims to investigate the safety, tolerability, and preliminary clinical efficacy of BEN301 Injection in patients with autoimmune diseases.
- Conditions
- Systemic Sclerosis (SSc), Rheumatoid Arthritis (RA), Sjogren's Syndrome (SS), Idiopathic Inflammatory Myopathies(IIM)
- Trial name
- NCT07527936
- Testing
- Single administration of 1x10^8 viable CD4+ CAR+ Foxp3+ cells., Single administration of 3x10^8 viable CD4+ CAR+ Foxp3+ cells.
- Where
- China
- Volunteers
- 24 people
- Sponsor
- RenJi Hospital
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.
- Trial name
- NCT04632953
- Testing
- No Intervention
- Where
- 16 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Systemic Lupus Erythematosus, Inflammatory Myopathy
UCAR T-cell Therapy Targeting CD19/ BCMA(QT-019C) in Patients With Relapse/ Refractory Autoimmune Diseases
This is an investigator-initiated trial to evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells（QT-019C） in With Relapse/Refractory Autoimmune Diseases.
- Trial name
- NCT07410377
- Testing
- UCAR-T cells
- Where
- China
- Sponsor
- The Affiliated Hospital of Xuzhou Medical University
Muscle Weakness, Critical Illness Myopathy
CPAx: Responsiveness and Minimal Clinically Important Difference
Intensive care unit (ICU) acquired weakness is a common complication associated with long-term physical impairments in survivors of a critical illness.
- Conditions
- Muscle Weakness, Critical Illness Myopathy, Critical Illness Polyneuropathy, Critical Illness Polyneuromyopathy
- Trial name
- NCT06419699
- Where
- 3 sites in Australia, Switzerland
- Volunteers
- 120 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Behcet's Disease, Idiopathic Inflammatory Myopathies
Drug Rediscovery for Rare Immune Mediated Inflammatory Diseases
Research into novel therapies for rare, immune-mediated inflammatory diseases (IMIDs) is limited due to small patient populations.
- Conditions
- Behcet's Disease, Idiopathic Inflammatory Myopathies, IgG4-related Disease
- Trial name
- DRIMID (NCT06285539)
- Testing
- Filgotinib
- Where
- 6 sites in Netherlands
- Volunteers
- 60 people
- Sponsor
- UMC Utrecht
Multiple Sclerosis (MS), Neuromyelitis Optica Spectrum Disorder (NMOSD)
Safety and Pharmacodynamics of QH103 Cell Injection in the Treatment of Patients With Relapsed/Refractory Antibody-Mediated Neurological Autoimmune Diseases.
This study is an open-label, exploratory, prospective clinical trial with dose escalation(according to "3+3" design), to evaluate the safety and tolerability of QH103(Universal CD19 CAR-γδT Cell Injection)in the treatment of…
- Conditions
- Multiple Sclerosis (MS), Neuromyelitis Optica Spectrum Disorder (NMOSD), Autoimmune Encephalitis (AE), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Trial name
- NCT07526493
- Testing
- Universal CD19 CAR-γδT Cell Injection, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Tongji Hospital
Critical Illness Myopathy
NLRP3 Inflammasome and Physical Therapy in ICU-Acquired Weakness
The goal of this clinical trial is to study whether physical therapy can reduce NLRP3 inflammasome activation and muscle atrophy in patients with critical illness myopathy (CIM).
- Trial name
- PT-NLRP3-CIM (NCT07017517)
- Testing
- Motomed Letto® servo-assisted cycling + standard physiotherapy, Standard physiotherapy
- Where
- 3 sites in Chile
- Volunteers
- 24 people
- Sponsor
- University of Chile
Systemic Sclerosis (SSc), Idiopathic Inflammatory Myopathy (IIM)
Phase 1 Study of HBI0101 CAR-T in Refractory B-Cell Autoimmune Diseases
A Phase 1 study of HBI0101 BCMA-CART in B-Cell Mediated Autoimmune Rheumatic Diseases. The goal of the study is evaluation of safety and identification of the maximum HBI0101 CART dose that may be administered safely to patients…
- Conditions
- Systemic Sclerosis (SSc), Idiopathic Inflammatory Myopathy (IIM), Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE)
- Trial name
- NCT07085676
- Testing
- HBI0101 CART
- Where
- Israel
- Volunteers
- 120 people
- Sponsor
- Polina Stepensky
Rheumatoid Arthritis, Spondylitis, Ankylosing
Recombinant Herpes Zoster Vaccine in Patients With Autoimmune Rheumatic Diseases
Introduction: Patients with autoimmune rheumatic diseases (ARDs), rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), psoriatic arthritis (PAs), ankylosing spondylitis (AS), systemic lupus erythematosus (SLE), primary…
- Conditions
- Rheumatoid Arthritis, Spondylitis, Ankylosing, Spondyloarthritis, Systemic Lupus Erythematosus
- Trial name
- RZVRheum (NCT05879419)
- Testing
- Recombinant Herpes Zoster Vaccine (RZV), MTX Discontinuation, MMF Discontinuation
- Where
- Brazil
- Volunteers
- 2,005 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
Muscular Dystrophies, Congenital Myopathy
Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular Disorders
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a programmable ambulation exoskeleton (KeeogoTM Dermoskeleton System, B-Temia Inc., Quebec, Canada) in patients with…
- Conditions
- Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies, Mitochondrial Myopathies
- Trial name
- Exo-KGO1 (NCT05199246)
- Testing
- Lower-limb powered dermoskeleton
- Where
- France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Muscular Dystrophies, Congenital Myopathy
Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular Disorders
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a powered knee-hip dermoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland) in patients with neuromuscular disorders, ii)…
- Conditions
- Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies, Mitochondrial Myopathies
- Trial name
- Exo-NMD1 (NCT05200702)
- Testing
- Knee-hip Powered Soft Exoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland)
- Where
- France
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
SLE - Systemic Lupus Erythematosus, IIM- Idiopathic Inflammatory Myopathies
A Study of CC312 for Relapsed/Refractory Autoimmune Diseases
This study is an open-label, multiple ascending dose investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of CC312 in adult patients with relapsed or…
- Conditions
- SLE - Systemic Lupus Erythematosus, IIM- Idiopathic Inflammatory Myopathies, SSc-Systemic Sclerosis
- Trial name
- NCT07193810
- Testing
- CC312
- Where
- China
- Volunteers
- 6 people
- Sponsor
- CytoCares Inc
Nemaline Myopathy
Natural History Study for Patients With Nemaline Myopathy in the UK
The goal of this study is to to learn more about what assessments would be useful to measure for NM and what normally happens during the lives of people with NM to support future clinical trial development.
- Trial name
- NatHis-NM-MDUK (NCT06670378)
- Where
- 4 sites in United Kingdom
- Volunteers
- 45 people
- Sponsor
- University of Oxford
Adenylosuccinate Synthase 1 Deficient Myopathy
A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of ASA-001 in two adults diagnosed with ADSS1 deficient myopathy.
- Trial name
- (ASA-CS01) (NCT07412821)
- Testing
- adenylosuccinic acid
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Cure ADSSL1
Statin Adverse Reaction, Statin-induced Myopathy
Metabolic and Mitochondrial Changes in Statin-Induced Myopathy
The goal of this observational study is to learn whether statin-related muscle symptoms are linked to problems in fat metabolism in muscle cells, specifically involving acylcarnitines, in adults taking or not taking statins.
- Trial name
- NCT07511686
- Testing
- Blood sampling and laboratory assessment, Muscle biopsy
- Where
- Latvia
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Riga Stradins University
IBMPFD
Natural History Study of Patients With VCP-related Disease
A natural history study to understand the expected progression of disease in patients with confirmed mutations in the VCP gene over 1 year.
- Trial name
- NCT04823143
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Nationwide Children's Hospital