Clinical trials: Myopathies
Low Back Pain, Musculoskeletal Pain
Connective Tissue Motion Measure 2
This study aims to compare the Connective Tissue Motion Measure (CTMM) in low back pain and no-low back pain subjects, evaluate the reliability of the CTMM, and examine the correlation of CTMM with low back pain symptoms…
- Trial name
- NCT01784094
- Testing
- StromaGlide
- Where
- United States
- Volunteers
- 195 people, healthy volunteers welcome
- Sponsor
- Stromatec, Inc.
Temporomandibular Disorders, Pain
Nasal Carbon Dioxide (CO2)for the Treatment of Temporomandibular Disorders (TMD)Related Pain
This purpose of this study is to evaluate the safety and efficacy of treatment with nasal CO2 in the treatment of pain and other symptoms related to temporomandibular disorder (TMD).
- Conditions
- Temporomandibular Disorders, Pain, Headaches
- Trial name
- NCT00484029
- Testing
- Nasal Carbon Dioxide, Air
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Capnia, Inc.
Fibromyalgia
A Safety and Efficacy Study of Xyrem® in Subjects With Fibromyalgia
The objective of this trial is to evaluate the safety and efficacy of Xyrem® compared to placebo for the treatment of fibromyalgia in a randomized, double blind, placebo controlled, parallel group trial.
- Trial name
- NCT00371137
- Testing
- Xyrem®
- Where
- 70 sites in United States
- Volunteers
- 548 people
- Sponsor
- Jazz Pharmaceuticals
Duchenne Muscular Dystrophy
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who…
- Trial name
- NCT03218995
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis
Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)
The goal of this clinical trial is to assess the efficacy and safety of Pimicotinib (ABSK021) in patients with Tenosynovial Giant Cell Tumor (TGCT).
- Conditions
- Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath
- Trial name
- NCT05804045
- Testing
- Pimicotinib(ABSK021)
- Where
- 40 sites in United States, Canada and 5 more
- Volunteers
- 94 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
Temporomandibular Disorder, Orofacial Pain
An Open Label Study Assessing REN Treatment in Headache Management in Temporomandibular Disorders
Temporomandibular disorders (TMD) encompass a group of conditions involving the masticatory muscles, temporomandibular joint (TMJ), and associated structures.
- Trial name
- NCT07639944
- Testing
- Nerivio treatment
- Where
- 4 sites in United States
- Volunteers
- 100 people
- Sponsor
- Theranica
Duchenne Muscular Dystrophy
A Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT02246478
- Testing
- TAS-205
- Where
- Japan
- Volunteers
- 23 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Dupuytren's Contracture
CORRECT: COmmunity RegistRy Study Evaluating Dupuytren's Contracture Treatment
This observational study will prospectively collect data on treatment options, patient/investigator satisfaction, requirement for retreatment or additional treatment, and long-term outcomes of 3 treatment modalities(XIAFLEX…
- Trial name
- CORRECT (NCT01715467)
- Where
- 27 sites in United States
- Volunteers
- 162 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Age-related Muscle Decline
CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy
This Phase 1/2a, open-label, non-randomized study is designed to evaluate the safety and tolerability of intramuscular AAV9-Follistatin gene therapy administered either as monotherapy or in combination with a VEGF-encoding…
- Trial name
- CALM-AF-AI (NCT07443826)
- Testing
- AAV9-Follistatin gene therapy, VEGF Plasmid
- Where
- Honduras
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Unlimited Biotechnology LLC
Rotator Cuff Tendinopathy
Clinical Benefits and Safety of Tropho Tend in the Management of Painful Rotator Cuff Tendinopathy
This is a prospective, open label, controlled, exploratory clinical investigation aimed to evaluate the clinical benefit and safety of Tropho Tend in the conservative management of painful Rotator Cuff Tendinopathy (RCT).
- Trial name
- NCT06809543
- Testing
- Polynucleotides (Tropho Tend), Rehabilitation physiotherapy
- Where
- Italy
- Volunteers
- 60 people
- Sponsor
- Mastelli S.r.l
Blepharospasm, Torticollis
MDs on Botox Utility (MOBILITY)
The MOBILITY Project is a prospective, non-randomized, observational, multi-centre evaluation of Health Utility via the SF-12® Health Survey Scores and the SF-6D in patients receiving BOTOX® for therapeutic use.
- Conditions
- Blepharospasm, Torticollis, Strabismus, Muscle Spasticity
- Trial name
- NCT00535938
- Testing
- botulinum toxin type A
- Where
- Canada
- Volunteers
- 1,372 people
- Sponsor
- Allergan
ADHD, Fibromyalgia
TD-9855 Mass Balance Study
The purpose of the study is to evaluate the absorption, distribution, metabolism, and excretion (ADME) of a single oral dose of [14C]TD-9855
- Trial name
- NCT01924143
- Testing
- TD-9855
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Theravance Biopharma
Tenosynovial Giant Cell Tumor
MOVE: Tenosynovial Giant Cell Tumor (TGCT) Real-World Participant Experience With Vimseltinib
The goal of this observational study is to better understand real-world use of vimseltinib, treatment patterns, and health-related quality of life in participants treated with vimseltinib for TGCT.
- Trial name
- MOVE (NCT07075471)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Deciphera Pharmaceuticals, LLC
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.
Idiopathic Inflammatory Myopathy, Dermatomyositis
RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
- Conditions
- Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy
- Trial name
- NCT06154252
- Testing
- CABA-201 following preconditioning with fludarabine and cyclophosphamide
- Where
- 38 sites in United States, Germany and 2 more
- Volunteers
- 74 people
- Sponsor
- Cabaletta Bio
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Spasticity, Multiple Sclerosis
A Study With IPX056 in Subjects With Spasticity Associated With Multiple Sclerosis
To explore the safety and efficacy of IPX056 compared with baclofen tablets for alleviation of symptoms of spasticity associated with multiple sclerosis (MS).
- Trial name
- NCT00914290
- Testing
- IPX056, Baclofen IR
- Where
- 11 sites in United States
- Volunteers
- 23 people
- Sponsor
- Impax Laboratories, LLC
GNE Myopathy, Hereditary Inclusion Body Myopathy
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA).
- Trial name
- GNE Myopathy (NCT01517880)
- Testing
- Sialic Acid Extended Release (SA-ER)
- Where
- 4 sites in United States, Israel
- Volunteers
- 46 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
TGCT
Study of Emactuzumab for Tenosynovial Giant Cell Tumor (TGCT)
This is a multicenter, Phase 3, randomised, double-blind, placebo-controlled study, which aims to evaluate the efficacy and safety of the investigational drug emactuzumab for the treatment of patients with localized or diffuse…
- Trial name
- TANGENT (NCT05417789)
- Testing
- Emactuzumab
- Where
- 33 sites in United States, Belgium and 10 more
- Volunteers
- 128 people
- Sponsor
- SynOx Therapeutics Limited