Clinical trials: Myopathies
Systemic Lupus Erythematosus, Systemic Sclerosis
CAR-T Cells in Systemic B Cell Mediated Autoimmune Disease
The investigational product is designed to effectively combat B cells in patients with autoimmune diseases. Autologous T cells enriched with CD4/CD8 are genetically engineered using a lentiviral vector to express chimeric antigen…
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis, Dermatomyositis, Polymyositis
- Trial name
- CASTLE (NCT06347718)
- Testing
- anti-CD19 CAR T cell therapy
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Miltenyi Biomedicine GmbH
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
- Trial name
- ENDEAVOR (NCT04626674)
- Testing
- delandistrogene moxeparvovec
- Where
- 12 sites in United States
- Volunteers
- 83 people
- Sponsor
- Sarepta Therapeutics, 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.
Spasticity, Multiple Sclerosis
Evaluate the Maintenance of Effect After Long-term Treatment With Sativex® in Subjects With Symptoms of Spasticity Due to Multiple Sclerosis
The purpose of this study is to evaluate the maintenance of effect after long-term treatment with Sativex® in subjects with symptoms of spasticity due to Multiple Sclerosis (MS) who have been receiving long-term benefit from…
- Trial name
- NCT00702468
- Testing
- Sativex
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- Jazz Pharmaceuticals
Duchenne Muscular Dystrophy
A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy
- Trial name
- NCT04587908
- Testing
- TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort]
- Where
- 5 sites in Japan
- Volunteers
- 104 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Spinal Cord Injury, Muscle Spasticity
Safety and Efficacy of Oral Fampridine-Sustained Release (SR) for the Treatment of Spasticity Resulting From Spinal Cord Injury
Normally, nerve fibers carry electrical impulses through the spinal cord, providing communication between the brain and the arms and legs.
- Trial name
- NCT01683838
- Testing
- Fampridine-SR
- Where
- 30 sites in United States, Canada
- Volunteers
- 204 people
- Sponsor
- Acorda Therapeutics
Autoimmune Diseases, Systemic Lupus Erythematosus
A Study to Test CRT-402 in Refractory Autoimmune Disease
Patients with refractory autoimmune diseases often have limited treatment options and ongoing disease activity despite standard therapies.
- Conditions
- Autoimmune Diseases, Systemic Lupus Erythematosus, Systemic Scleroderma, Myositis
- Trial name
- NCT07778446
- Testing
- CRT-402
- Where
- Australia
- Volunteers
- 34 people
- Sponsor
- Myeloid Therapeutics
Myositis
A Study to Understand How the Study Medicine (PF-06823859) Works in People With Active Idiopathic Inflammatory Myopathies [Dermatomyositis (DM) and Polymyositis (PM)]
The purpose of the study is to understand how the study medicine PF-06823859 (dazukibart) works in people with idiopathic inflammatory myopathies (DM and PM).
- Trial name
- NCT05895786
- Testing
- PF-06823859
- Where
- 138 sites in United States, Argentina and 18 more
- Volunteers
- 318 people
- Sponsor
- Pfizer
Stroke, Spasticity
Phase III Study of Botulax® to Treat Post Stroke Upper Limb Spasticity
The purpose of this study is to evaluate the safety and efficacy of Botulax® compared to Botox® reducing upper limb muscle tone in post stroke patients.
- Trial name
- NCT01915459
- Testing
- Botulinum toxin type A(Botulax®), Botulinum toxin type A(Botox®)
- Where
- 6 sites in South Korea
- Volunteers
- 186 people
- Sponsor
- Hugel
Physical Activity, Muscle Recovery
Effect of NMN on Muscle Recovery and Physical Capacity in Healthy Volunteers With Moderate Physical Activity
The purpose of this study is to evaluate the effect of NMN supplementation (250 and 500 mg/day over 38 days) compared to placebo in healthy volunteers with moderate physical activity on muscle recovery, physical capacity…
- Trial name
- NCT04664361
- Testing
- Nicotinamide mononucleotide
- Where
- France
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Seneque SA
X-Linked Myotubular Myopathy
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness.
- Trial name
- EXCEL (NCT06581146)
- Testing
- No Intervention
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Astellas Gene Therapies
Fibromyalgia
Duloxetine Versus Placebo in the Treatment of FMS
To test the hypothesis that the efficacy and safety of duloxetine has beneficial effects of the reduction of pain severity as measured by the average pain item of the BPI and the PGI-I in patients with fibromyalgia syndrome.
- Trial name
- NCT00233025
- Testing
- Duloxetine
- Where
- 34 sites in United States, Germany and 4 more
- Volunteers
- 320 people
- Sponsor
- Eli Lilly and Company
Duchenne Muscular Dystrophy
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.
- Trial name
- NCT01826474
- Testing
- PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week
- Where
- 6 sites in Belgium, France and 3 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Tenosynovial Giant Cell Tumor
Study of Pimicotinib in Japanese Participants With Tenosynovial Giant Cell Tumor (TGCT) (J-MANEUVER)
The primary purpose of this study is to assess the tolerability, pharmacokinetics, and efficacy of pimicotinib in Japanese participants with TGCT
- Trial name
- NCT07499362
- Testing
- Pimicotinib
- Where
- 4 sites in Japan
- Volunteers
- 20 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Pigmented Villonodular Synovitis, PVNS
MCS110 in Patients With Pigmented Villonodular Synovitis (PVNS)
This study, designed as a proof of concept study of MCS110 in pigmented villonodular synovitis, assessed the clinical response to MCS110 treatment in Pigmented Villonodular Synovitis (PVNS) patients, after a single or multiple…
- Conditions
- Pigmented Villonodular Synovitis, PVNS, Giant Cell Tumor of the Tendon Sheath, GCCTS
- Trial name
- NCT01643850
- Testing
- MCS110
- Where
- 8 sites in United States, Switzerland
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Stroke, TBI
Characterization of the DELPhI System in Assessing Brain's Functionality in Different Neurological Disorders
We use Transcranial magnetic stimulation (TMS), combined with simultaneous registration of electroencephalograph (EEG),for examining human cortical functionality.
- Conditions
- Stroke, TBI, Fibromyalgia, PDD
- Trial name
- NCT03832413
- Testing
- DELPhI (TMS-EEG analysis)
- Where
- Israel
- Volunteers
- 193 people, healthy volunteers welcome
- Sponsor
- QuantalX Neuroscience
Muscle Strength, Muscle Weakness
Performance Output With Haptics - Evaluating Athlete Response and Unrealized Potential
This minimal risk, randomized, double-blind, placebo controlled Institutional Review Board (IRB)-approved observational study with functional measurements, will evaluate athletic performance after use of a drug- free…
- Trial name
- POWER-UP (NCT06430879)
- Testing
- VICTORY PATCH
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- SuperPatch Limited LLC
Duchenne Muscular Dystrophy
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Trial name
- NCT03373968
- Testing
- Givinostat
- Where
- 39 sites in United States, Belgium and 9 more
- Volunteers
- 206 people
- Sponsor
- Italfarmaco
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX…
- Trial name
- NCT03943290
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 62 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals