Clinical trials: Atrophy
Duchenne Muscular Dystrophy
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle.
- Trial name
- NCT01610440
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Inflammatory Bowel Diseases, Crohn Disease
Nutrition and Clinical Outcomes in IBD
The goal of this observational study is to demonstrate that nutritional status and body composition have an impact on clinical outcomes in inflammatory bowel disease (IBD). The main objectives are: 1.
- Conditions
- Inflammatory Bowel Diseases, Crohn Disease, Ulcerative Colitis, Malnutrition
- Trial name
- NUTRICO-IBD (NCT06550310)
- Where
- United Kingdom
- Volunteers
- 300 people
- Sponsor
- Barts & The London NHS Trust
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)
Background: Myotonic dystrophy is a long-term genetic disorder that affects muscle function. Symptoms include gradually worsening muscle loss and weakness. Muscles often contract and cannot relax.
- Trial name
- NCT03867435
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
Stem Cell Therapy in Duchenne Muscular Dystrophy
The purpose of this study was to study the effect of stem cell therapy in patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT02241434
- Testing
- Stem Cell
- Where
- India
- Sponsor
- Neurogen Brain and Spine Institute
Muscular Dystrophy, Duchenne, DMD
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
- Trial name
- NCT04337112
- Testing
- viltolarsen
- Sponsor
- NS Pharma, Inc.
Lasers, Atrophy of Vagina
The Use of Laser in the Treatment of Atrophic Vulvovaginitis
This study evaluates the efficacy and security of laser for atrophy treatment. Half of participants will receive the laser treatment and the other half placebo.
- Conditions
- Lasers, Atrophy of Vagina, Atrophy Vulva
- Trial name
- NCT04297319
- Testing
- Laser diiodo
- Where
- Argentina
- Volunteers
- 20 people
- Sponsor
- Hospital Italiano de Buenos Aires
Duchenne Muscular Dystrophy
Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research…
- Trial name
- NCT00468832
- Where
- 21 sites in United States, Argentina and 6 more
- Volunteers
- 551 people, healthy volunteers welcome
- Sponsor
- Cooperative International Neuromuscular Research Group
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Duchenne Muscular Dystrophy
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
- Trial name
- NCT04240314
- Testing
- scAAV9.U7.ACCA
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Megan Waldrop
Alveolar Clefts, Alveolar Bone Grafting
Evaluation of Using Iliac Crest With and Without Platelet-Rich Fibrin in Repair of Congenital Alveolar Cleft
A randomized clinical trial aims to evaluate the clinical and radiographic outcomes of the repair of congenital alveolar clefts using Iliac crest graft with and without mixing with platelet-rich fibrin
- Conditions
- Alveolar Clefts, Alveolar Bone Grafting, Alveolar Bone Loss
- Trial name
- NCT06924216
- Testing
- alveolar bone grafting, alveolar bone grafting Plus Platelet rich fibrin
- Where
- Egypt
- Volunteers
- 16 people
- Sponsor
- Suez Canal University
Muscular Dystrophies
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876…
- Trial name
- NCT01803412
- Testing
- Drisapersen
- Where
- 13 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- BioMarin Pharmaceutical
Healthy, Muscular Dystrophy
Studies of Patients With Skin Disease, Patients With Neurological Degenerations, and Normal Volunteers
It is proposed that patients with skin disease due to presumed immunologic, genetic or viral-induced abnormalities, patients with neurological degenerations, and normal controls be evaluated with various in vitro studies of…
- Conditions
- Healthy, Muscular Dystrophy, Retinal Degeneration, Skin Diseases
- Trial name
- NCT00001164
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Progeria
Clinical Studies of Progeria
This study will examine children with Hutchinson-Gilford Progeria syndrome, a genetic disease that causes many changes to the body over time, including heart disease, bone changes, hair loss, and joint and skin changes.
- Trial name
- NCT00094393
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Muscular Dystrophies
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
- Trial name
- NCT01153932
- Testing
- GSK2402968
- Where
- 13 sites in Australia, Belgium and 7 more
- Volunteers
- 53 people
- Sponsor
- GlaxoSmithKline
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)
Cerebral Palsy, Spina Bifida
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Muscular Dystrophies, Musculoskeletal Disorder
- Trial name
- NCT03851107
- Testing
- Engagement in 6-week community-based activity program
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- McGill University
Inflammatory Myopathy, Duchenne Muscular Dystrophy
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
The aim of our study is to Assess skeletal muscle structural status in children with inflammatory myositis and Duchenne muscular dystrophy using musculoskeletal ultrasound and to perform a longitudinal follow up of these changes…
- Trial name
- NCT03786913
- Testing
- Quantitative muscle ultrasound measurements
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Benha University
Sarcopenia, COPD
Evaluate the Effect of Dietary Supplement Combined Exercise Among Patients With Sarcopenia Comorbid With Lung Disease
This trial is a prospective multi-center study. The purpose of this study is to evaluate the effect of dietary supplement combined with supervised exercised on the physical performance, body composition and lung function among…
- Conditions
- Sarcopenia, COPD, ILD
- Trial name
- NCT05926713
- Testing
- Sarcojoint®, supervised exercise
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- Orient Europharma Co., Ltd.
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.