Clinical trials: MYOPATHY
Duchenne Muscular Dystrophy
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S.
- Trial name
- NCT02592941
- Testing
- Deflazacort
- Where
- 42 sites in United States
- Sponsor
- PTC Therapeutics
Tenosynovial Giant Cell Tumor
A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan
This phase 2, multicenter, two-part, open-label, single-arm study will be conducted in Japan and will evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of pexidartinib in adult participants with symptomatic…
- Trial name
- NCT04703322
- Testing
- Pexidartinib
- Where
- 6 sites in Japan
- Volunteers
- 9 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Low Back Pain, Radicular Syndrome
Comparison of High-Intensity and Conventional Physical Therapy in Musculoskeletal Disorders
This pilot randomized controlled trial aims to compare the clinical effectiveness of advanced high-intensity physical therapy modalities with conventional physical therapy in patients suffering from musculoskeletal disorders.
- Conditions
- Low Back Pain, Radicular Syndrome, Sacroiliac Joint Dysfunction, Shoulder Impingement Syndrome
- Trial name
- NCT07903142
- Testing
- Repetitive Peripheral Magnetic Stimulation (rPMS), High-Intensity Laser Therapy (HIL), Extracorporeal Shockwave Therapy (ESWT)
- Where
- Czechia
- Volunteers
- 60 people
- Sponsor
- BTL Industries Ltd.
Mitochondrial Myopathy
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate…
- Trial name
- MMPOWER (NCT02367014)
- Testing
- elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose)
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- Stealth BioTherapeutics Inc.
Fibromyalgia
Study Of A Controlled Release Formulation Of Pregabalin In Fibromyalgia Patients
The purpose of this study is to evaluate the efficacy and safety of a controlled release formulation of pregabalin administered once daily as compared to placebo in the treatment of fibromyalgia.
- Trial name
- NCT01271933
- Testing
- pregabalin
- Where
- 52 sites in United States, Canada and 2 more
- Volunteers
- 441 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or…
- Trial name
- NCT04812106
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Joint Pain, Musculoskeletal Pain
IDEA-033 Open Label Study
The main aim of the study is to evaluate safety, efficacy, compliance, and usage of epicutaneously applied IDEA-033 in joint / musculoskeletal pain or soft tissue inflammation.
- Conditions
- Joint Pain, Musculoskeletal Pain, Stiffness, Soft Tissue Inflammation in Designated Target Area(s)
- Trial name
- NCT00372333
- Testing
- IDEA-033
- Where
- Germany
- Volunteers
- 491 people
- Sponsor
- IDEA AG
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Dermatomyositis
An Extension Study for Participants Who Have Completed the Treatment Period of a Qualifying Parent Study
The purpose of this research study is to evaluate the long-term safety, and tolerability of PF-06823859 study drug in adult participants with Dermatomyositis (DM) from a qualifying study.
- Trial name
- NCT05192200
- Testing
- Anti-Beta Interferon (PF-06823859)
- Where
- 25 sites in United States, Hungary and 2 more
- Volunteers
- 24 people
- Sponsor
- Pfizer
Fibromyalgia
A Study of Duloxetine in Fibromyalgia
The purpose of the study is to assess the effectiveness and safety of duloxetine in participants with fibromyalgia.
- Trial name
- NCT01552057
- Testing
- Duloxetine 60 mg
- Where
- Japan
- Volunteers
- 393 people
- Sponsor
- Eli Lilly and Company
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Blepharospasm, Hemifacial Spasm
Post Marketing Surveillance Study of Dysport
The purpose of this study is to provide further information regarding the risks and benefits of Dysport in marketed indications.
- Conditions
- Blepharospasm, Hemifacial Spasm, Cervical Dystonia, Spasmodic Torticollis
- Trial name
- NCT00210431
- Where
- 36 sites in France, Germany and 1 more
- Volunteers
- 783 people
- Sponsor
- Ipsen
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.
Atrophy, Muscular
31P MRS Ischaemic Exercise Optimisation and COPD
An MRI study to develop a reliable methodology for 31P MRS ischaemic exercise in order to obtain a consistent standard of measurement of muscle metabolism while maintaining an acceptable level of subject comfort and use optimised…
- Trial name
- NCT01476072
- Testing
- MRI scan
- Where
- United Kingdom
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Myotonic Dystrophy
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES…
- Trial name
- NCT06523400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 7 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.
Non Ruptured Tendon Injuries, Tendinopathy
Tissue Repair Device (VergenixTM STR) in Patients With Epicondylitis (Tennis Elbow)
The VergenixTM STR device is intended for the repair of non ruptured tendon injuries (Tendinopathy).
- Trial name
- CP-STR-01 (NCT02309307)
- Testing
- Tissue Repair Device (VergenixTM STR)
- Where
- 3 sites in Israel
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Collplant
Duchenne Muscular Dystrophy
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
- Trial name
- NCT02251600
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Multiple Sclerosis, Spasticity, Muscle
Performance and Long-term Safety of FlowOx2.0™, Multiple Sclerosis, Spasticity and Pain
This study is based on a 4-week double-blind, randomized, controlled, parallel design investigation to investigate the impact of intermittent negative pressure on spasticity and pain in people with multiple sclerosis (pwMS)…
- Conditions
- Multiple Sclerosis, Spasticity, Muscle, Pain
- Trial name
- NCT05598736
- Testing
- FlowOx 2.0
- Where
- Norway
- Volunteers
- 58 people
- Sponsor
- Otivio AS
X-linked Myotubular Myopathy
A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger.
- Trial name
- INCEPTUS (NCT02704273)
- Where
- 8 sites in United States, Canada and 3 more
- Volunteers
- 34 people
- Sponsor
- Astellas Gene Therapies
Duchenne Muscular Dystrophy
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon…
- Trial name
- NCT02530905
- Testing
- SRP-4045
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.