Clinical trials: Atrophy
Leber's Hereditary Optic Neuropathy
Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study
Expanded Access Program for Idebenone in Patients with Leber's Hereditary Optic Neuropathy who completed the LEROS Study
- Trial name
- US EAP LHON (NCT04381091)
- Testing
- Idebenone 150 MG Oral Tablet
- Sponsor
- Santhera Pharmaceuticals
Muscular Dystrophy, Duchenne
Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD) have changes in strength when given the drug Pentoxifylline as a rescue treatment.
- Trial name
- NCT00243789
- Testing
- Pentoxifylline
- Where
- 11 sites in United States, Argentina and 4 more
- Volunteers
- 64 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Genetic Disease, Communication
The Role of Family Functioning in Promoting Adaptation in Siblings of Individuals With Duchenne Muscular Dystrophy (DMD)
Background: We want to learn more about the relationship between the way families function and how children adapt to having a sibling with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01386515
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Muscular Dystrophy, Duchenne
Pentoxifylline in Duchenne Muscular Dystrophy
In this study, the primary aim will be to estimate the magnitude and variability of strength change over time that may be expected for subjects on the study treatment.
- Trial name
- NCT00102453
- Testing
- Pentoxifylline
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Duchenne Muscular Dystrophy
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and…
- Trial name
- NCT07172737
- Testing
- Respiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Izmir Katip Celebi University
Atopic Dermatitis
Opzelura Experience Study
Atopic dermatitis is a common multifactorial skin disease. Topicals are the mainstay of treatment, but adherence to topicals is often abysmal, possibly worsened by the complexity of recommended treatment algorithms.
- Trial name
- NCT07661979
- Testing
- ruxolitinib, ruxolitinib and an Eczema Action Plan
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Wake Forest University Health Sciences
Diabetes Mellitus, Iron Metabolism Disorders
Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy
Patients who are genetically diagnosed with the recently reported and rare Wolfram syndrome type 2 ( WFS2) and have the degenerative and symptomatic disease including signs such as diabetes, platelet aggregation defect or visual…
- Conditions
- Diabetes Mellitus, Iron Metabolism Disorders, Gastroduodenal Ulcer, Optic Atrophy
- Trial name
- NCT02882477
- Testing
- Deferiprone, Acetylcysteine, Sitagliptin and Metformin
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Hadassah Medical Organization
Cardiomyopathy, Hypertrophic, Muscular Dystrophy, Oculopharyngeal
Study of Muscle Abnormalities in Patients With Specific Genetic Mutations
Hypertrophic cardiomyopathy (HCM) is a genetically inherited disease affecting the heart. It causes thickening of heart muscle, especially the chamber responsible for pumping blood out of the heart, the left ventricle.
- Trial name
- NCT00001871
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystrophies
Taking part in community activities is essential for health and well-being. Yet, it is highly restricted for young people with Duchenne and Becker Muscular Dystrophies (DBMD), especially as they grow into adulthood.
- Trial name
- NCT06540365
- Testing
- Pathways and Resources for Engagement and Participation (PREP)
- Volunteers
- 6 people
- Sponsor
- University of Haifa
Duchenne Muscular Dystrophy
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT1-EDO51 (NCT06079736)
- Testing
- PGN-EDO51
- Where
- 5 sites in Canada
- Volunteers
- 7 people
- Sponsor
- PepGen Inc
Multiple System Atrophy
Trial of Autologous Mesenchymal Stem Cells in Patients With Multiple System Atrophy
This study is based on positive results in open label trial of mesenchymal stem cells therapy in patients with Multiple System Atrophy (MSA).
- Trial name
- NCT00911365
- Testing
- autologous mesenchymal stem cells, normal saline
- Where
- South Korea
- Volunteers
- 27 people
- Sponsor
- Yonsei University
Muscular Dystrophy, Arthrogryposis
WREX Outcome Study
This study is to evaluate how the Wilmington Robotic Exoskeleton (WREX) is working for children who are using the WREX, or have used it in the past.
- Conditions
- Muscular Dystrophy, Arthrogryposis, Spinal Muscular Atrophy
- Trial name
- NCT02218593
- Testing
- WREX orthosis
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic
Duchenne Muscular Dystrophy (DMD)
DMD and Gamified Physiotherapy
Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular disease that limits children's physical function, mobility, and participation in daily life.
- Trial name
- Gamified DMD (NCT07368400)
- Testing
- Gamified Mobile Physiotherapy Exercise Application, Standard Home Exercise Instructions
- Volunteers
- 46 people
- Sponsor
- Başak Çağla Arslan
Muscular Atrophy, Spinal
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
- Trial name
- Rainbowfish (NCT03779334)
- Testing
- Risdiplam
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 26 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Tremor, Involuntary Movements
Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen
Hyperkinetic movement disorders in patients with diseases of motor neurons will be studied. Patients with spinal muscular atrophy (SMA) and motor neuron disease patients will be studied.
- Conditions
- Tremor, Involuntary Movements, SMA, MND (Motor Neurone DIsease)
- Trial name
- NCT04825119
- Testing
- Nusinersen
- Where
- Slovenia
- Volunteers
- 110 people
- Sponsor
- University Medical Centre Ljubljana
SMA II
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
- Trial name
- NCT05901987
- Testing
- GC101
- Where
- 5 sites in China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Myotonic Dystrophy Type 1
Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy
Study to focus on the defining and managing the neuropsychological abnormalities of myotonic dystrophy and to find out if the neuropsychological abnormalities have any correlation with changes seen on Magnetic Resonance Imaging.
- Trial name
- CHRI (NCT02269865)
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Stanford University
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