Clinical trials: Myopathy
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
Muscle Disorder, Musculoskeletal Diseases
HIFEM for Musculoskeletal System Improvement
The goal of this clinical trial is to investigate the effect of the BTL-899 device on improving the Musculoskeletal System in healthy adult volunteers.
- Trial name
- NCT06677086
- Testing
- Treatment with BTL-899
- Where
- 3 sites in United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- BTL Industries Ltd.
Rhabdomyolysis, Renal Failure
Myoglobin Removal by High Cut-off CVVHD
A high cut-off dialyzer (septeX) is tested for the removal of myoglobin which is not efficiently removed by standard high flux dialysis membranes.
- Trial name
- HicoRhabdo (NCT01467180)
- Testing
- septeX, HF CVVH
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- Vantive Health LLC
Limb Girdle Muscular Dystrophy
A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
- Trial name
- NCT05906251
- Testing
- SRP-6004
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sarepta Therapeutics, Inc.
Polymyositis, Dermatomyositis
Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy
Trial to Evaluate the Efficacy and Safety of Abatacept subcutaneous (SC) in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy
- Conditions
- Polymyositis, Dermatomyositis, Autoimmune Necrotizing Myopathy, Overlap Myositis
- Trial name
- NCT02971683
- Testing
- Abatacept subcutaneous
- Where
- 79 sites in United States, Australia and 9 more
- Volunteers
- 149 people
- Sponsor
- Bristol-Myers Squibb
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
X-Linked Myotubular Myopathy
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition.
- Trial name
- ASPIRO (NCT03199469)
- Testing
- Resamirigene bilparvovec
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 27 people
- Sponsor
- Astellas Gene Therapies
Duchenne Muscular Dystrophy
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT03532542
- Testing
- Casimersen, Golodirsen
- Where
- 50 sites in United States, Belgium and 11 more
- Volunteers
- 171 people
- Sponsor
- Sarepta Therapeutics, Inc.
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Facioscapulohumeral Muscular Dystrophy, FSHD
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Fibromyalgia
Efficacy and Tolerability of CELERGEN in Fibromyalgia
Celergén® is a dietary supplement with anti-inflammatory and antioxidant properties composed of marine sperm extract, hydrolyzed collagen, CoQ10, lutein and selenium.
- Trial name
- NCT03911882
- Testing
- Celergen Administration
- Where
- Spain
- Volunteers
- 90 people
- Sponsor
- Celergan SA
Systemic Lupus Erythematosus (SLE), Systemic Sclerosis
Exploratory Clinical Study on YTS109 Cell Therapy for 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), including LN and SLE-ITP, Sjogren's Syndrome, etc.
- Conditions
- Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, Sjogren's Syndrome (SS), Inflammatory Myopathy
- Trial name
- NCT07236801
- Testing
- YTS109 cell
- Where
- 2 sites in China
- Volunteers
- 18 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Duchenne Muscular Dystrophy (DMD)
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
- Trial name
- NCT06867107
- Testing
- SAT-3247
- Where
- 6 sites in United States, Australia
- Volunteers
- 30 people
- Sponsor
- Satellos Bioscience, Inc.
Musculoskeletal Pain
Efficacy and Safety of the Combination of Ketoprofen and Cyclobenzaprine and Caffeine in Osteomuscular Treatment
The purpose of this study is to evaluate the safety and efficacy of an association with one anti-inflammatory and one muscle relaxant plus caffeine compared to one anti-inflammatory plus caffeine in the treatment of osteomuscular…
- Trial name
- NCT02862977
- Testing
- ketoprofen and cyclobenzaprine association with caffeine, Cyclobenzaprine with caffeine
- Where
- 6 sites in Brazil
- Volunteers
- 414 people
- Sponsor
- EMS
Epicondylitis, Lateral, Tendinopathy, Elbow
OVT for Epicondylosis (Tennis Elbow)
To assess the residual risk of OrthoVisc-T (OVT) for the treatment of chronic lateral epicondylosis (Tennis Elbow). This trial will provide clinical data on a subject population not previously researched which have failed prior…
- Trial name
- NCT04026191
- Testing
- OrthoVisc®-T (OVT)
- Where
- 3 sites in Czechia
- Volunteers
- 50 people
- Sponsor
- Anika Therapeutics, Inc.
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis
Study of Vimseltinib for Tenosynovial Giant Cell Tumor
This is a multicenter Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called vimseltinib for the treatment of tenosynovial giant cell tumor (TGCT) in cases where surgical removal of the…
- Conditions
- Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath, Tenosynovial Giant Cell Tumor, Diffuse
- Trial name
- MOTION (NCT05059262)
- Testing
- Vimseltinib
- Where
- 35 sites in United States, Australia and 11 more
- Volunteers
- 123 people
- Sponsor
- Deciphera Pharmaceuticals, LLC
Duchenne Muscular Dystrophy
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage…
- Trial name
- NCT02667483
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
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