Clinical trials: Myopathies
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite [UEC] score) as…
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
- Trial name
- GNEM (NCT02377921)
- Testing
- aceneuramic acid extended-release (Ace-ER)
- Where
- 13 sites in United States, Bulgaria and 5 more
- Volunteers
- 89 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Myotonic Dystrophy 1
Study of ATX-01 in Participants With DM1
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated.
- Trial name
- ArthemiR (NCT06300307)
- Testing
- ATX-01
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 56 people
- Sponsor
- ARTHEx Biotech S.L.
Dermatomyositis, Polymyositis
Study Evaluating Efficacy and Safety of Froniglutide (PF1801) in Patients With Idiopathic Inflammatory Myopathy
This is a Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Froniglutide in Patients With Idiopathic Inflammatory Myopathy ("FROniGlutide Study")
- Conditions
- Dermatomyositis, Polymyositis, Idiopathic Inflammatory Myopathies
- Trial name
- FROG (NCT05833711)
- Testing
- Froniglutide
- Where
- South Korea
- Volunteers
- 39 people
- Sponsor
- Immunoforge Co. Ltd.
Low Back Pain, Low Back Pain, Recurrent
Blue Light Device for Pain Therapy
The purpose of this study is to determine if the Pain Relief Patch, which shines light of a limited wavelength on the painful area of the back, relieves chronic musculoskeletal back pain.
- Conditions
- Low Back Pain, Low Back Pain, Recurrent, Musculoskeletal Pain, Chronic Pain
- Trial name
- PAINCT02 (NCT01528332)
- Testing
- Pain Relief Patch, Control PRP device
- Where
- 3 sites in Germany
- Volunteers
- 171 people
- Sponsor
- Philips Electronics Nederland BV
Primary Mitochondrial Disease
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
The primary objective of the FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease (PMD) following 48 weeks of treatment.
- Trial name
- FALCON (NCT05650229)
- Testing
- KL1333
- Where
- 55 sites in United States, Australia and 9 more
- Volunteers
- 180 people
- Sponsor
- Pharming Technologies B.V.
Tendinopathy
Achilles Tendon Repair With Bioinductive Implant
To evaluate bioinduction of new tissue and tendon healing after implantation of the Rotation Medical Bioinductive Implant used as adjunct to surgical repair in the treatment of insertional Achilles tendonitis.
- Trial name
- NCT02811003
- Testing
- Rotation Medical Bioinductive Implant
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Smith & Nephew, Inc.
Musculoskeletal Pain
Efficacy and Safety of the Combination of Ketoprofen and Cyclobenzaprine 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 agent compared to the one muscle relaxant agent isolated in the treatment of osteomuscular pain…
- Trial name
- NCT03025113
- Testing
- ketoprofen and cyclobenzaprine association, Cyclobenzaprine
- Where
- Brazil
- Volunteers
- 416 people
- Sponsor
- EMS
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
Childhood-onset Systemic Lupus Erythematous, ANCA-Associated Vasculitis (AAV)
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
- Conditions
- Childhood-onset Systemic Lupus Erythematous, ANCA-Associated Vasculitis (AAV), Juvenile Myasthenia Gravis, Juvenile Dermatomyositis
- Trial name
- NCT07089121
- Testing
- Descartes-08
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Cartesian Therapeutics
Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C.
- Conditions
- Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies, MELAS Syndrome, Mitochondrial Myopathies
- Trial name
- NCT03888716
- Testing
- KL1333
- Where
- 2 sites in United Kingdom
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Abliva AB
Cerebral Palsy, Spasticity
Dose-response Study of Efficacy and Safety of Botulinum Toxin Type A to Treat Spasticity of the Arm(s) or of Arm(s) and Leg(s) in Cerebral Palsy
The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of one or both arms alone or in combination with injections into one or both legs are effective and safe in treating…
- Trial name
- XARA (NCT02002884)
- Testing
- IncobotulinumtoxinA (8 Units per kg body weight), IncobotulinumtoxinA (6 Units per kg body weight), IncobotulinumtoxinA (2 Units per kg body weight)
- Where
- 32 sites in United States, Argentina and 4 more
- Volunteers
- 351 people
- Sponsor
- Merz Pharmaceuticals GmbH
Fibrodysplasia Ossificans Progressiva
A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP).
- Trial name
- ANDECAL (NCT06508021)
- Testing
- Andecaliximab
- Where
- 3 sites in United States
- Volunteers
- 92 people
- Sponsor
- Ashibio Inc
Compartment Syndrome
Phase I, Arteriocyte Magellan MAR01 Therapy - Compartment Syndrome and Battlefield Trauma
Compartment syndrome (CS) is a condition resulting from increased pressure within a compartment, which compromises circulation and can lead to critical limb ischemia.
- Trial name
- Magellan MAR01 (NCT01837264)
- Testing
- Magellan®
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Arteriocyte, Inc.
Fibromyalgia
CBD (Cannabidiol)/THC (Tetrahydrocannabinol) Solution as a Pharmacological Strategy for Patients With Fibromyalgia (FibroCann)
Fibromyalgia is considered a chronic pain syndrome, non-inflammatory, of unknown etiology, which manifests itself in the musculoskeletal system in up to 2.5% of the general population, predominantly in females, mainly between 35…
- Trial name
- NCT05283161
- Testing
- cannabidiol and tetrahydrocannabinol
- Volunteers
- 40 people
- Sponsor
- FG Brasil LTDA
Muscular Dystrophy, Facioscapulohumeral
Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1).
- Trial name
- NCT06131983
- Testing
- SRP-1001 for Injection
- Where
- 16 sites in Australia, Canada and 5 more
- Volunteers
- 60 people
- Sponsor
- Sarepta Therapeutics, Inc.
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.
Muscular Dystrophies
A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling
This is a Phase I, open-label, single-dose, 1-period study to compare EDG-5506 concentrations measured from venous and capillary blood sampling in healthy adult subjects.
- Trial name
- NCT05492734
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Compartment Syndrome
Assessment of Near Infrared Spectroscopy as a Diagnostic Tool in Acute Compartment Syndrome
The purpose of this study is to define the reliability and accuracy of Near Infrared Spectroscopy (NIRS) in the detection of intra-compartmental tissue perfusion in injured and noninjured extremities over time.
- Trial name
- NCT01077934
- Testing
- near-infrared spectroscopy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- J&M Shuler
Duchenne Muscular Dystrophy
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up.
- Trial name
- HOPE-OLE (NCT06304064)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Capricor Inc.
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