Clinical trials: Muscle Diseases
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.
Neuroblastoma, Medulloblastoma
Molecular-Guided Therapy for Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup.
- Conditions
- Neuroblastoma, Medulloblastoma, Glioma, Ependymoma
- Trial name
- NCT02162732
- Testing
- Guided Therapy
- Where
- 19 sites in United States, Lebanon
- Volunteers
- 186 people
- Sponsor
- Giselle Sholler
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
Hernias, Diaphragmatic, Congenital
Resistance Exercise in Children With Post-operative Congenital Diaphragmatic Hernia
Congenital diaphragmatic hernia (CDH) is a life-threatening condition with long-term complications including respiratory tract infections, respiratory muscle weakness, and abnormal lung functions.
- Trial name
- hernia (NCT04900649)
- Testing
- chest resistance exercise, chest expansion exercises
- Where
- Saudi Arabia
- Volunteers
- 32 people
- Sponsor
- Qassim University
Cerebral Palsy
Effects of PEDAL Program With and Without EMS On Mobility And Gait In Children With Cerebral Palsy
PEDAL is a program that focuses on juvenile endurance and limb building for kids with cerebral palsy. Studying how PEDAL with and without Electrical Muscle Stimulation (EMS) affects these metrics in an effort to determine whether…
- Trial name
- NCT06460311
- Testing
- PEDAL and EMS, PEDAL
- Where
- Pakistan
- Volunteers
- 22 people
- Sponsor
- Riphah International 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
Congenital Heart Disease, Telerehabilitation
The Effect of Telerehabilitation Exercises in Children With Congenital Heart Disease
This study aims to investigate the effects of functional inspiratory muscle training on physical performance and quality of life in pediatric patients with congenital heart disease.
- Conditions
- Congenital Heart Disease, Telerehabilitation, Functional Inspiratory Muscle Training
- Trial name
- CHD (NCT07854171)
- Testing
- Inspiratory Muscle Training, Functional Inspiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Izmir Democracy University
Spinal Muscular Atrophy
Spinal Muscular Atrophy Neonatal Screening Program
Spinal muscular atrophy (SMA) is a group of disorders caused by the degeneration of the motor neuron cells of the anterior horn of the spinal cord and, in some subtypes, of the bulbar motor neurons.
- Trial name
- NCT06310421
- Testing
- Screening SMA test
- Where
- 2 sites in Italy
- Volunteers
- 11,500 people
- Sponsor
- IRCCS Burlo Garofolo
Sarcopenia
Sarcopenia Physical Activity and Metabolomic
Muscle failure (sarcopenia or dynapenia) is a factor of frailty and therefore, ultimately, of loss of autonomy in the elderly. Currently, no biomarker of muscle failure has a high sensitivity, specificity and positive predictive…
- Trial name
- SPAM (NCT05199207)
- Testing
- Physical activity programm
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Nice
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
Prader-Willi Syndrome, Sarcopenia
Ultrasound to Assess Sarcopenia in Prader Willi Syndrome
The aim of the study is to examine the clinical applicability of ultrasound as a diagnostic tool for sarcopenia in individuals with PWS by investigating the association between ultrasound-derived measurements, DXA-derived…
- Conditions
- Prader-Willi Syndrome, Sarcopenia, Metabolic Syndrome
- Trial name
- PWS (NCT06448871)
- Testing
- Ultrasound assessment of the right lower extremity muscles, Dual-energy X-ray absorptiometry (DXA) scan
- Where
- Taiwan
- Volunteers
- 49 people
- Sponsor
- Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation
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
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Bladder Cancer
Urine-based Detection of Non-muscle Invasive Bladder
Non-muscle invasive bladder cancer (NMIBC), which comprises approximately 75% of bladder tumors, has the highest recurrence rate of all cancers, with around 70% of the patients developing local recurrences, despite elaborated…
- Trial name
- SOLUSION (NCT03962933)
- Where
- Denmark
- Volunteers
- 196 people
- Sponsor
- Nessn Azawi
Non Invasive Ventilation, Spinal Muscular Atrophy
Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
SMA (Spinal Muscular Atrophy) is a rare neuromuscular disease characterized by motoneuron damage. Symptoms consist of respiratory involvement with numerous respiratory infections and eventually respiratory failure, for which NIV…
- Conditions
- Non Invasive Ventilation, Spinal Muscular Atrophy, Respiratory Bacterial Infection
- Trial name
- AVNIR (NCT05712330)
- Testing
- non invasive ventilation samples, patient samples, parent questionnaire
- Where
- France
- Volunteers
- 8 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
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
Cerebral Palsy, Muscle Hypertonia
Evaluation of the Effect of the Presence Of Clowns on Pain and Anxiety Seen During Injections Botulinum Toxin in Child
In children requiring botulinum toxin injections, improving supervisory procedures of injection sessions to reduce pain and improve the experience of this invasive procedure is needed.
- Trial name
- CLOWN-TOX (NCT03149263)
- Testing
- distraction
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
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