Clinical trials: Atrophy
Duchenne Muscular Dystrophy
Dual Task in Duchenne Muscular Dystrophy
This study was planned to determine the effects of the dual-task performance of children with DMD with motor dysfunction and varying degrees of cognitive impairment compared to their healthy peers, to compare the dual-task…
- Trial name
- NCT05575648
- Testing
- 10 meter walk test
- Where
- Turkey (Türkiye)
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
- Trial name
- ENHANCE (NCT07542314)
- Testing
- ELEVIDYS, Sirolimus, Glucocorticoids
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Childhood Cancer
Effect of Nutrition Counseling and Physical Exercise Prescription on Body Composition and Nutritional Status of 5-18 Years Pediatric Cancer Patients on Treatment at Jimma Medical Center Pediatric Hematology and Oncology Unit(JMC-PHOU), Jimma, Ethiopia
Malnutrition and associated muscle wasting are common health problems among pediatric cancer patients. However, the role of dietary counseling and physical exercise prescription intervention on both nutritional status and muscle…
- Trial name
- JMC-PHOU (NCT07558486)
- Testing
- dietary counseling and physical exercise prescription
- Where
- Ethiopia
- Volunteers
- 104 people
- Sponsor
- Jimma University
Sepsis-Associated Encephalopathy
Role of MSCT Volumetry in Assessment of Brain Atrophy in Septic Patients
assessment of brain atrophy associated with septic ICU patients by using MSCT Volumetry
- Trial name
- NCT06112119
- Testing
- MSCT
- Volunteers
- 55 people
- Sponsor
- Assiut University
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
The Duchenne Registry
The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- NCT02069756
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- The Duchenne Registry
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and…
- Trial name
- ESSENCE (NCT02500381)
- Testing
- SRP-4045, SRP-4053
- Where
- 76 sites in United States, Argentina and 22 more
- Volunteers
- 228 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Flu Vaccine Study in Neuromuscular Patients 2011
The purpose of the study is to compare the immune response of two different injection methods (Intramuscular V.S. Subcutaneous) of the 2011-2012 seasonal Influenza (Flu) vaccine among patients with neuromuscular conditions who…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Congenital Muscular Dystrophy
- Trial name
- NCT01422200
- Testing
- 2011-2012 seasonal flu vaccine Subcutaneous, 2011-2012 seasonal flu vaccine Intramuscular
- Volunteers
- 22 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neuromuscular Diseases, Duchenne Muscular Dystrophy
Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dystrophy
For ambulatory children with DMD, physiotherapy is aimed at protecting ambulation, improving motor performance to the best level and increasing quality of life.
- Conditions
- Neuromuscular Diseases, Duchenne Muscular Dystrophy, Dystrophy, Dystrophy, Muscular
- Trial name
- NCT03507530
- Testing
- Assessments
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Canan İpek
Duchenne Muscular Dystrophy
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
Canakinumab is an anti-interleukin 1 beta (IL1β) antibody approved for use in young children with familial Mediterranean fever, systemic onset juvenile idiopathic arthritis and TNF-receptor associated periodic fever syndrome.
- Trial name
- NCT03936894
- Testing
- Canakinumab Injection [Ilaris]
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's National Research Institute
REM Behavior Disorder, Parkinsonism
Efficacy and Tolerability of Ramelteon in Patients With Rapid Eye Movement (REM) Behavior Disorder and Parkinsonism
Parkinson's disease (PD) is the second most common neurodegenerative disorder of the elderly that affects a million patients in US. Sleep dysfunction impacts up to 90% of PD patients.
- Trial name
- NCT00745030
- Testing
- Rozerem
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Northwestern University
Duchenne Muscular Dystrophy
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment…
- Trial name
- ENDURE (NCT06270719)
- Testing
- Delandistrogene Moxeparvovec, Standard of Care
- Where
- 29 sites in United States
- Volunteers
- 500 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
This single center open-label pilot study will enroll 15 non-ambulatory children with Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical cardiomyopathy (defined as a cardiac ejection fraction…
- Trial name
- NCT02964377
- Testing
- (+)- Epicatechin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Craig McDonald, MD
Vulvar Atrophy, Vaginal Atrophy
Efficacy and Safety Of Spil's Estradiol Vaginal Tablet
Estradiol vaginal tablet is a tablet which hydrates upon contact with moisture, releasing 17ß-estradiol. The estradiol in estradiol vaginal tablet is chemically and biologically identical to the endogenous human estradiol and is…
- Trial name
- NCT01753102
- Testing
- Estradiol
- Where
- India
- Volunteers
- 49 people
- Sponsor
- Sun Pharmaceutical Industries Limited
Duchenne Muscular Dystrophy
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne…
- Trial name
- NCT01396239
- Testing
- AVI-4658 (Eteplirsen)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Cardiac Outcome Measures in Children With Muscular Dystrophy
The purpose of the research study is to evaluate different cardiac measures that are obtained by echocardiographic tests in patients with muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01066455
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Acne Scars - Atrophic
Nano Fat Injection With or Without Fractional Carbon Dioxide Laser in Acne Scars
Assessment of nano fat grafting combined with carbon dioxide laser resurfacing versus nano fat grafting alone in treating facial atrophic acne scars
- Trial name
- NCT05985265
- Testing
- Fat grafting
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Assiut University
Neurological Regression, Myoclonus
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis.
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Trial name
- NCT00029965
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Muscle Weakness, Problems Moving Their Arms and Legs
Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults
People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and legs.
- Trial name
- NCT06998134
- Testing
- Extension assist knee ankle foot orthosis (EA-KAFO)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
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
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