Clinical trials: Neuromuscular Diseases
Dermatomyositis
A Study Evaluating the Effects of GLPG3667 Given as Oral Treatment for up to 24 Weeks in Adults With Dermatomyositis
The purpose of this study is to evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered GLPG3667 once daily for 24 weeks in adult participants with dermatomyositis…
- Trial name
- GALARISSO (NCT05695950)
- Testing
- GLPG3667
- Where
- 54 sites in United States, Argentina and 13 more
- Volunteers
- 40 people
- Sponsor
- Lakefront Biotherapeutics NV
Neuralgia
Non-Interventional Study In Patients With Peripheral Or Central Neuropathic Pain Treated With Lyrica
The purpose of this study is to obtain more data about efficacy and safety of the study drug Lyrica.
- Trial name
- NCT00541476
- Volunteers
- 746 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Malnutrition, Sarcopenia
Evaluation of an Oral Nutritional Supplement Containing AN777
To evaluate the effect of an experimental oral nutritional supplement containing AN777 compared to another oral nutritional supplement in a malnourished, elderly population.
- Trial name
- NCT01191125
- Testing
- medical food with AN777, oral nutritional formula
- Where
- 23 sites in United States, Belgium and 6 more
- Volunteers
- 331 people
- Sponsor
- Abbott Nutrition
Amyotrophic Lateral Sclerosis (ALS)
Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral…
- Trial name
- NCT04577404
- Testing
- MT-1186
- Where
- 44 sites in United States, Canada and 4 more
- Volunteers
- 124 people
- Sponsor
- Shionogi
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-3 (NCT05126758)
- Testing
- Deramiocel (CAP-1002)
- Where
- 20 sites in United States
- Volunteers
- 106 people
- Sponsor
- Capricor Inc.
Diabetic Neuropathies
A Study Comparing the Effectiveness and Safety of Tramadol HCl/Acetaminophen Versus Placebo for the Treatment of Painful Neuropathy in Diabetic Patients
The purpose of this study is to explore the pain-reieiving effects and safety of tramadol HCl/acetaminophen as compared to placebo in patients experiencing painful diabetic neuropathy.
- Trial name
- NCT00210847
- Testing
- tramadol, acetaminophen
- Volunteers
- 313 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Amyotrophic Lateral Sclerosis
Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
The study is to determine the feasibility, safety, toxicity, and maximum tolerated (safe) dose of human spinal derived neural stem cell transplantation for the treatment of Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT01730716
- Testing
- Human spinal cord stem cell implantation
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neuralstem Inc.
Fibromyalgia
Safety Study of DS-5565 for Treatment of Fibromyalgia Pain in Subjects With Chronic Kidney Disease
DS-5565 (mirogabalin) is being studied as treatment for fibromyalgia (FM) pain. Because it is excreted through the kidneys, people who have reduced kidney function will not process the drug as well as with those with normal…
- Trial name
- NCT02496884
- Testing
- DS-5565
- Where
- 39 sites in United States, Bulgaria and 6 more
- Volunteers
- 56 people
- Sponsor
- Daiichi Sankyo
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
ALS (Amyotrophic Lateral Sclerosis)
Ciprofloxacin/Celecoxib Combination in Patients With ALS
This is an open label, off label study, to provide interested ALS patients with Ciprofloxacin/Celecoxib fixed dose combination, while assessing safety and tolerability, routine disease progression measures (ALSFRS-R and Vital…
- Trial name
- NCT04090684
- Testing
- Fixed dose combination Ciprofloxacin/Celecoxib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- NeuroSense Therapeutics Ltd.
Acellular Pertussis, Diphtheria
Immunogenicity and Reactogenicity of a Booster Dose of GSK Bio's DTPa-HBV-IPV/Hib Vaccine
The purpose of this booster study is to evaluate, in subjects primed in the primary study 106786, the persistence, at the time of the booster vaccination, of antibodies elicited by the different formulation of DTPa-HBV-IPV/ Hib…
- Conditions
- Acellular Pertussis, Diphtheria, Poliomyelitis, Haemophilus Influenzae Type b
- Trial name
- NCT00627458
- Testing
- Infanrix Hexa
- Where
- 6 sites in Finland
- Volunteers
- 403 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Congenital Myasthenic Syndrome
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7…
- Trial name
- NCT06078553
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 152 people
- Sponsor
- argenx
Myasthenia Gravis, Generalized
Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis
The Polish multicentre observational (non-interventional) study aiming to collect data on the management and clinical outcomes of patients with gMG that received ravulizumab.
- Trial name
- MG-ARCADIA (NCT06909253)
- Testing
- Ravulizumab
- Where
- 11 sites in Poland
- Volunteers
- 50 people
- Sponsor
- AstraZeneca
Dermatomyositis
Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis
This is a Phase 3 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of dermatomyositis.
- Trial name
- DETERMINE (NCT03813160)
- Testing
- Lenabasum 20 mg, Lenabasum 5 mg
- Where
- 54 sites in United States, Bulgaria and 11 more
- Volunteers
- 176 people
- Sponsor
- Corbus Pharmaceuticals Inc.
Generalized Myasthenia Gravis
A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the effectiveness, safety and tolerability of zilucoplan auto-injector (ZLP-AI) self-administration.
- Trial name
- NCT06471361
- Testing
- Zilucoplan
- Where
- 11 sites in United States, Poland and 1 more
- Volunteers
- 31 people
- Sponsor
- UCB Biopharma SRL
Diabetic Neuropathies, Insomnia Due to Medical Condition
To Assess the Efficacy of Investigational Product on the Neuropathic Pain in Individuals Suffering From Type II Diabetes Mellitus.
this study is to be carried out to examine the effect of Investigation product on diabetic individuals suffering from diabetic neuropathic pain.
- Trial name
- NCT04504305
- Testing
- NeuropAWAY, Microcystalline cellulose
- Where
- 2 sites in India
- Volunteers
- 64 people
- Sponsor
- Vedic Lifesciences Pvt. Ltd.
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
Neuropathic Pain
Non Invasive Brain Evaluation and Treatment for Neuropathic Pain
This trial is designed to preliminary evaluate the efficacy and safety of NIBS system for evaluation and treatment of neuropathic pain.
- Trial name
- NIBSNP (NCT02740062)
- Testing
- NIBS system for evaluation and non invasive current stimulation
- Where
- Israel
- Volunteers
- 30 people
- Sponsor
- NIBS NeuroScience Technologies
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