Clinical trials: Muscle Disorders
Cystic Fibrosis, Bronchiectasis
Comparison of the Efficacy of Comprehensive Respiratory Physiotherapy in Children With Cystic Fibrosis and Non-Cystic Fibrosis Bronchiectasis
The effect of comprehensive respiratory physiotherapy applications on respiratory function, functional capacity and peripheral muscle strength in children with cystic fibrosis and non-cystic fibrosis will be compared.
- Trial name
- NCT04170114
- Testing
- Comprehensive Respiratory Physiotherapy
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Bezmialem Vakif University
Congenital Disorders
Hypertrophic Myopathy in Children
The purpose of this study is to identify the patients seen in our practice who are seen with Hypertrophic Myopathy diagnosis in order to better understand the presenting characteristics of their disease, the diagnostic testing to…
- Trial name
- NCT00267462
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Children's Healthcare of Atlanta
Obesity (Disorder)
The Effectiveness of Respiratory Muscle Training and Exercise in Obese Adolescents
This study aims to investigate the effects of respiratory muscle training and exercise on respiratory muscle strength, pulmonary function, and exercise tolerance in obese adolescents diagnosed with specific learning disabilities.
- Trial name
- NCT07787546
- Testing
- Respiratory Muscle Training, Exercise
- Where
- Turkey (Türkiye)
- Volunteers
- 44 people
- Sponsor
- Medipol University
Spinal Muscular Atrophy
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
- Trial name
- NCT05824169
- Testing
- GC101
- Where
- 4 sites in China
- Volunteers
- 18 people
- Sponsor
- GeneCradle Inc
Rehabilitation
Early Rehabilitation in Critical Illness Survivors
Background: Few studies showed that early rehabilitation on the Intensive Care Unit (ICU) results in a better functional outcome at hospital discharge and reduced hospital stay.
- Trial name
- ER (NCT02754505)
- Testing
- Early rehabilitation, Usual care
- Where
- Austria
- Volunteers
- 53 people
- Sponsor
- Danube Hospital
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Cardiac Magnetic Resonance in Children With Muscular Dystrophy
This protocol will exploit novel state of the art cardiovascular magnetic resonance techniques to examine important changes in the heart in children with muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01081080
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Spinal Muscular Atrophy
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age.
- Trial name
- NCT00227266
- Testing
- Valproic Acid and Levocarnitine
- Where
- 6 sites in United States, Canada
- Volunteers
- 94 people
- Sponsor
- University of Utah
Sleep Disturbance, Postmenopausal Symptoms
Effect of Sleep Restriction on Adipose Tissue and Skeletal Muscle Insulin Sensitivity
Inadequate sleep is an independent risk factor for metabolic abnormalities (such as obesity, insulin resistance, and hyperglycemia).
- Conditions
- Sleep Disturbance, Postmenopausal Symptoms, Insulin Sensitivity, Cardiovascular Risk Factor
- Trial name
- SLEEP-IN (NCT04286451)
- Testing
- Sleep Restriction, Habitual Sleep
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Pennington Biomedical Research Center
Smooth Muscle Dysfunction Syndrome (SMDS)
Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
The purpose of this study is to determine whether SMDS patients treated with NR at the proposed dose exhibit decreased glucose uptake in the aorta, to determine if NR treatment results in measurable changes of blood NAD+ and NR…
- Trial name
- NCT06280482
- Testing
- Nicotinamide riboside (NR)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- The University of Texas Health Science Center, Houston
MELAS Syndrome
Nitric Oxide Production in MELAS Syndrome
Introduction Baylor College of Medicine and Texas Children's Hospital are recruiting individuals with MELAS syndrome for a clinical study.
- Trial name
- NCT01339494
- Testing
- Arginine and citrulline supplementations
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Pediatrics, Muscle Spasticity
BOTOX® Treatment in Pediatric Upper Limb Spasticity
This study will evaluate the safety and efficacy of BOTOX® (botulinum toxin Type A) in pediatric patients with upper limb spasticity.
- Conditions
- Pediatrics, Muscle Spasticity, Cerebral Palsy, Stroke
- Trial name
- NCT01603602
- Testing
- botulinum toxin Type A
- Where
- 45 sites in United States, Canada and 7 more
- Volunteers
- 235 people
- Sponsor
- Allergan
Urinary Incontinence
Effects of Action Observation Training (AOT) in the Achievement of Urinary Continence in Men After Robot-assisted Laparoscopic Prostatectomy (RALP)
Urinary incontinence (UI) is one of the most common side effects of radical prostatectomy (RP) and compromises men's quality of life.
- Trial name
- NCT05311267
- Testing
- Action observation training
- Where
- Italy
- Volunteers
- 92 people
- Sponsor
- Istituto Clinico Humanitas
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
The goal of this observational study is to investigate whether the type, location, and extent of pathogenic variants in the DMD gene are associated with cardiac dysfunction in male children, adolescents, and young adults with…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Cardiomyopathy
- Trial name
- NCT07515235
- Where
- Greece
- Volunteers
- 65 people
- Sponsor
- Aristotle University Of Thessaloniki
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Duchenne Muscular Dystrophy
Safety and Efficacy of P-188 NF in DMD Patients
This is an open-label study to evaluate the safety, tolerability and efficacy of daily, subcutaneous dosing with P-188 NF (Carmeseal-MD™) in non-ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03558958
- Testing
- P-188 NF
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Phrixus Pharmaceuticals, Inc.
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
Duchenne Muscular Dystrophy (DMD)
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
- Trial name
- NCT06900049
- Testing
- LE051
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Congenital Myopathies, Muscular Dystrophy
Personalized Disease Modeling for Rare Genetic Myopathies
The goal of this observational study is to learn how rare genetic muscle diseases change over time and whether muscle cells grown from a participant's blood can help researchers understand the disease.
- Trial name
- NCT07914049
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Akron Children's Hospital
Cerebral Palsy (CP)
Constraint-induced Movement Therapy Combined With Functional Electrical Stimulation in Children With Cerebral Palsy
This randomized controlled trial aims to compare the effectiveness of Constraint-Induced Movement Therapy (CIMT) combined with Functional Electrical Stimulation (FES) versus CIMT alone in improving upper limb function, reducing…
- Trial name
- CIMT-FES-CP (NCT07587476)
- Testing
- Constraint-Induced Movement Therapy Combined with Functional Electrical Stimulation, Constraint-Induced Movement Therapy
- Where
- Pakistan
- Volunteers
- 46 people
- Sponsor
- Montiha Azeem
Brain and Central Nervous System Tumors, Gastrointestinal Stromal Tumor
Vaccine Therapy and Sargramostim in Treating Patients With Sarcoma or Brain Tumor
RATIONALE: Vaccines may make the body build an immune response to kill tumor cells. Colony-stimulating factors such as sargramostim increase the number of immune cells found in bone marrow or peripheral blood.
- Conditions
- Brain and Central Nervous System Tumors, Gastrointestinal Stromal Tumor, Sarcoma
- Trial name
- NCT00069940
- Testing
- sargramostim, telomerase: 540-548 peptide vaccine
- Where
- United States
- Sponsor
- Dana-Farber Cancer Institute
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