Clinical trials: Myopathy
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.
Duchenne Muscular Dystrophy, Performance
Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance
The investigator investigated the effect of lower limb flexibility on functional performance of children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03534349
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Post-COVID-19 Condition, Ulcerative Colitis
Emerging Indications for Hyperbaric Oxygen Treatment
This study will evaluate the effectiveness of hyperbaric oxygen therapy (HBOT) on treating emerging indications (i.e., conditions that have shown to potentially benefit from HBOT) using the Multicenter Registry for Hyperbaric…
- Conditions
- Post-COVID-19 Condition, Ulcerative Colitis, Crohn Disease, Calciphylaxis
- Trial name
- NCT07240649
- Testing
- Hyperbaric oxygen treatment (HBOT), 100% Oxygen
- Volunteers
- 100 people
- Sponsor
- Jay C. Buckey Jr.
Cerebral Palsy, Contracture
Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy
In this controlled dose-escalation study, we will study the initial safety, biological properties, and potential efficacy of 5-azacytidine (AZA).
- Trial name
- NCT06377085
- Testing
- 5-Azacytidine 10mg/m^2
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Shirley Ryan AbilityLab
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.
Muscular Dystrophy, Duchenne
Spironolactone Versus Prednisolone in DMD
This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy.
- Trial name
- NCT03777319
- Testing
- Spironolactone, Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Juvenile Primary Fibromyalgia Syndrome (JPFS), Fibromyalgia
Safety and Efficacy of Fluoxetine in Juvenile Fibromyalgia
The purpose of this research is to conduct an open, pilot trial to assess the efficacy and safety of fluoxetine in the treatment of Juvenile Primary Fibromyalgia Syndrome (JPFS).
- Trial name
- NCT00115804
- Testing
- Fluoxetine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Cincinnati
Fibromyalgia, Physiotherapy
Effectiveness of the Strain Counterstrain Technique in Women With Fibromyalgia Syndrome
This study evaluates whether strain counterstrain therapy affects pain, fatigue, psychological status, and quality of life in women diagnosed with fibromyalgia.
- Conditions
- Fibromyalgia, Physiotherapy, Pain, Strain Counterstrain Technique
- Trial name
- NCT07421609
- Testing
- Conservative Physiotherapy Program, Strain Counterstrain Therapy
- Where
- Turkey (Türkiye)
- Volunteers
- 105 people
- Sponsor
- Hasan Kalyoncu University
Peripheral Arterial Disease
Mechanisms That Produce the Leg Dysfunction of Claudication & Treatment Strategies
Intermittent claudication afflicts 5% of the US population older than 55 years of age and develops along with hardening of the arteries of the legs.
- Trial name
- NCT01970332
- Testing
- Revascularization Surgery, Supervised exercise therapy
- Where
- United States
- Volunteers
- 220 people
- Sponsor
- University of Nebraska
Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1)
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and…
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- NCT06809049
- Testing
- Music and movement education
- Where
- Canada
- Volunteers
- 13 people
- Sponsor
- Hanns Lochmuller
X-linked Myotubular Myopathy
Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)
This study is a longitudinal study evaluating the severity and progression of respiratory muscle function in patients with X-Linked Myotubular Myopathy (XLMTM) aged 0-14.
- Trial name
- NCT02453152
- Testing
- History and physical, Tidal breathing, Maximal respiratory pressures
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of Florida
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Musculoskeletal Pain, Work Related Stress
Physiatrist Ergonomic Intervention on Work Related Musculoskeletal Pain in Surgeons
The goal of this randomized control trial is to learn if physical therapy coaching and education improves work-related muscle pain in surgeons more than education alone. The main focuses of this study are to: 1.
- Conditions
- Musculoskeletal Pain, Work Related Stress, Quality of Life, Grip
- Trial name
- NCT05946018
- Testing
- Educational video on ergonomics, Verbal physical therapy coaching sessions
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- University of Texas Southwestern Medical Center
GNE Myopathy, GNE Related Diseases
A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
Background: GNE Myopathy is a disease that causes walking difficulties and increasing muscle weakness. It usually develops in young adults (between 20 and 30 years of age), and affects arm and leg muscles.
- Trial name
- NCT01417533
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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.
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
This is an open-label, single-arm, dose-escalation trial to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular…
- Trial name
- NCT07282652
- Testing
- RAG-18
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Peking Union Medical College Hospital
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 1
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Trial name
- NCT04583917
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 377 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Dystonia, Drug Induced Dystonia
Clinical Validation of DystoniaNet Deep Learning Platform for Diagnosis of Isolated Dystonia
This research involves retrospective and prospective studies for clinical validation of a DystoniaNet deep learning platform for the diagnosis of isolated dystonia.
- Conditions
- Dystonia, Drug Induced Dystonia, Parkinson Disease, Essential Tremor
- Trial name
- NCT05317390
- Testing
- DystoniaNet-based diagnosis of isolated dystonia
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Massachusetts Eye and Ear Infirmary
Interstitial Pneumonitis, Polymyositis
Investigation in Myositis-associated Pneumonitis of Prednisolone And Concomitant Tacrolimus
The purpose of the study is to evaluate the efficacy and safety of the combination treatment of tacrolimus and corticosteroid in polymyositis/dermatomyositis patients with interstitial pneumonitis with comparison against…
- Conditions
- Interstitial Pneumonitis, Polymyositis, Dermatomyositis
- Trial name
- IMPPACT (NCT00504348)
- Testing
- Tacrolimus
- Where
- 11 sites in Japan
- Volunteers
- 25 people
- Sponsor
- Tokyo Medical and Dental University
Duchenne Muscular Dystrophy
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
The purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.
- Trial name
- NCT00451074
- Testing
- Gentamicin infusions twice a week for six months
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Nationwide Children's Hospital
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