Clinical trials: Myopathies
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta 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.
Fibromyalgia
Pregabalin In Adolescent Patients With Fibromyalgia
The main purpose of this study is to evaluate the long-term safety of pregabalin in adolescent patients who participated in the previous fibromyalgia study (A0081180) and who wish to recieve open-label pregabalin
- Trial name
- NCT01020526
- Testing
- pregabalin
- Where
- 26 sites in United States, Czechia and 1 more
- Volunteers
- 63 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Dupuytren's Contracture
AA4500 (XIAFLEX™, Proposed Name) in the Treatment of Dupuytren's Contracture
This was a Phase 3, double-blind, randomized, placebo-controlled study conducted in the United States. Subjects with a diagnosis of Dupuytren's contracture in a metacarpophalangeal (MP) or proximal interphalangeal (PIP) joint…
- Trial name
- CORD-I (NCT00528606)
- Testing
- collagenase clostridium histolyticum
- Where
- 15 sites in United States
- Volunteers
- 308 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Rhabdomyolysis
Extracorporeal Therapy for the Removal of Myoglobin Using the CytoSorb in Patients With Rhabdomyolysis
Prospective, randomized non-blinded, controlled study to assess the feasibility of the CytoSorb as an adjunct to the standard of care in patients with rhabdomyolysis requiring renal replacement therapy.
- Trial name
- NCT02111018
- Testing
- CytoSorb Device, CVVH
- Where
- United States
- Sponsor
- CytoSorbents, Inc
Spasticity, Muscle
Validation of the Spasticity Related Quality of Life Questionnaire
The purpose of the study is to describe the psychometric properties (validity, reliability and responsiveness) of the SQOL-6D instrument in the context of routine clinical treatment of upper limb spasticity.
- Trial name
- SQOL-6D (NCT03442660)
- Testing
- Data collection
- Where
- 8 sites in United Kingdom
- Volunteers
- 104 people
- Sponsor
- Ipsen
Temporomandibular Disorders
Treatment Outcomes for Temporomandibular Disorders (TMD) Via the Clayton Intra-aural Device (CID) Clinical Trial
The purpose of this study is to characterize the safety profile and assess the effectiveness of the CID in treating subjects with temporomandibular disorders (TMDs).
- Trial name
- NCT00815776
- Testing
- Clayton Intra-aural Device (CID), Mouth Splint, Exercise Group
- Where
- Mexico
- Volunteers
- 152 people
- Sponsor
- Ascentia Health, Inc.
Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathy
A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases (Breakfree-1)
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases (Breakfree-1).
- Conditions
- Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathy, Systemic Sclerosis, Rheumatoid Arthritis
- Trial name
- NCT05869955
- Testing
- CC-97540, Fludarabine, Cyclophosphamide
- Where
- 46 sites in United States, Belgium and 4 more
- Volunteers
- 270 people
- Sponsor
- Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
Fibromyalgia
Fibromyalgia Response With Esreboxetine Evaluated Using a Randomized Withdrawal Research Design
The study is a Phase 3, double-blind, placebo-controlled, randomized withdrawal study to assess the efficacy and safety of AXS-14 in the management of fibromyalgia.
- Trial name
- FORWARD (NCT07398417)
- Testing
- AXS-14 (Esreboxetine)
- Where
- 53 sites in United States
- Volunteers
- 620 people
- Sponsor
- Axsome Therapeutics, Inc.
Fibromyalgia
A Study Comparing Duloxetine and Placebo in the Treatment of Fibromyalgia
The purpose of this study is to confirm the efficacy and safety of duloxetine 60-120 mg once daily in comparison to placebo on symptom improvement in patients meeting criteria for fibromyalgia aged 18 and older.
- Trial name
- NCT00673452
- Testing
- duloxetine hydrochloride
- Where
- 46 sites in United States, Puerto Rico
- Volunteers
- 530 people
- Sponsor
- Eli Lilly and Company
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
Polymyalgia Rheumatica
Efficacy and Safety of Olokizumab in Patients With Polymyalgia Rheumatica (PROMISE)
The primary objective of the study is to evaluate the efficacy of olokizumab (OKZ) 64 mg administered subcutaneously every 2 weeks compared with placebo in participants with polymyalgia rheumatica (PMR).
- Trial name
- NCT07670546
- Testing
- Olokizumab
- Where
- 20 sites in Russia
- Volunteers
- 125 people
- Sponsor
- R-Pharm International, LLC
Tennis Elbow
Efficacy and Safety of TenoMiR in Lateral Epicondylitis
This study is testing a drug called TenoMiR that is being developed for the treatment of tennis elbow (lateral epicondylitis). The study drug is a new compound that works by improving the quality of the collagen which helps…
- Trial name
- NCT06192927
- Testing
- TenoMiR, 0.9% Sodium Chloride Injection
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 123 people
- Sponsor
- Causeway Therapeutics
RYR-1 Myopathy
S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
This study proposes to test S 48168 (ARM210) in a Phase 1 trial in RYR1-RM patients, specifically. The objectives of this study are to explore the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD)/target…
- Trial name
- NCT04141670
- Testing
- S48168
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- RyCarma Therapeutics, Inc.
Muscle Soreness
A Study to Evaluate the Analgesic Properties of Topically Applied Menthol in Marathon Participants
The purpose of the research study is to better understand the factors that impact pain experience in adult (18+ years) marathon runners participating in the 2026 Chicago Marathon and to better understand if topically applied…
- Trial name
- NCT07873242
- Testing
- Observational, Non-Interventional Study
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Reckitt Benckiser Healthcare (UK) Limited
Chronic Pain, Anxiety
Nu-V3 Non-Invasive Nerve Stimulation Device Trial for Chronic Pain, Anxiety, Depression, Sleeplessness
The Nu-V3 Clinical Study is a prospective, single-arm, open-label, multi-center study using the Nu-V3 cranial nerve stimulation treatment device in patients with chronic pain, anxiety, depression, and/or sleeplessness.
- Conditions
- Chronic Pain, Anxiety, Depression, Sleeplessness
- Trial name
- Nu-V3 (NCT05335980)
- Testing
- Nu-V3
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Nu-Life Solutions
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Giant Cell Tumors
Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project
TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with…
- Trial name
- TOP (NCT02948088)
- Where
- 15 sites in United States, Austria and 6 more
- Volunteers
- 183 people
- Sponsor
- Daiichi Sankyo