Clinical trials: Congenital Myopathy
Muscular Dystrophy
Heart Imaging in Children With Muscular Dystrophy
Background: Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function.
- Trial name
- NCT01633242
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Facioscapulohumeral Muscular Dystrophy (FSHD)
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.
- Trial name
- FSHD (NCT02603562)
- Testing
- ATYR1940
- Where
- 7 sites in United States, France and 1 more
- Volunteers
- 8 people
- Sponsor
- aTyr Pharma, Inc.
Duchenne Muscular Dystrophy, Exon 44
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44, DMD
- Trial name
- NCT05996003
- Testing
- NS-089/NCNP-02
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Spinal Cord Injury, Spinal Cord Involvement
Clinical Trial of a Serious Game for Individuals With SCI/D
This study will evaluate the efficacy of a newly developed serious game, SCI HARD, to enhance self-management skills, self-reported health behaviors, and quality of life among adolescents and young adults with spinal cord injury…
- Conditions
- Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis
- Trial name
- NCT02341950
- Testing
- SCI Hard
- Where
- United States
- Volunteers
- 184 people
- Sponsor
- University of Michigan
Arthritis, Rheumatoid, Activities of Daily Living
Adaptation of EDAQ Into Turkish, Examining The Reliability and Validity For People With RA
The aim of the study is to adapt the Evaluation of Daily Activity Questionnaire into Turkish language, and to examine the reliability and validity for people with rheumatoid arthritis.
- Trial name
- NCT05244824
- Testing
- Adaptation into Turkish, Examining The Reliability and Validity for People with RA
- Where
- Turkey (Türkiye)
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy
This study investigates the correlation between assessments measuring functional capacity and functional capability in patients with Duchenne muscular dystrophy.
- Trial name
- NCT05249361
- Testing
- no intervention
- Where
- 2 sites in South Korea
- Volunteers
- 48 people
- Sponsor
- Samsung Medical Center
Duchenne Muscular Dystrophy
Evaluation of Home Based Assessments on Participants With DMD
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
- Trial name
- NCT05657938
- Testing
- Wearable Device, Duchenne Video Assessment (DVA)
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Solid Biosciences 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
Muscular Dystrophies
LARA: Use of JACO2 Controlled by Joystick or by Voice Control System, in Muscular Dystrophy
As part of the EMPATIA @ Lecco project, task 2.9 and task 4.5 respectively provide for the refinement and testing of robotic solutions on the market oriented towards patient empowerment.
- Trial name
- NCT04313049
- Testing
- JACO2, VOCAL CONTROL
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- IRCCS Eugenio Medea
Muscular Dystrophy, Duchenne
An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the nutritional supplement Coenzyme Q10 when added to steroids as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033189
- Testing
- Coenzyme Q10
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Muscle Diseases
Acceptance and Commitment Therapy for Muscle Disease
In adults, muscle diseases are usually chronic long-term conditions that do not have a definitive cure. Supportive care has been shown to reduce complications from muscle disease and improved survival in some cases.
- Trial name
- ACTMuS (NCT02810028)
- Testing
- Acceptance and Commitment Therapy (ACT)
- Where
- United Kingdom
- Volunteers
- 155 people
- Sponsor
- King's College Hospital NHS Trust
Upper Extremity Dysfunction
Movement Enhancing Device for Children
The purpose of this study is to assess: 1) how infants move their bodies to explore, play, and solve problems, 2) if this is different in a high-risk group of infants, and 3) if these abilities can be advanced in a high-risk…
- Trial name
- NCT01959581
- Testing
- Movement Enhancing Device
- Where
- 3 sites in United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Delaware
Muscular Dystrophy, Duchenne Muscular Dystrophy
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
- Trial name
- BMMNC (NCT01834040)
- Testing
- Intralesional/ Intravenous of Autologous Stem cells.
- Where
- India
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Chaitanya Hospital, Pune
Muscular Dystrophy, Duchenne
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically…
- Trial name
- MoveDMD® (NCT02439216)
- Testing
- Edasalonexent
- Where
- 5 sites in United States
- Volunteers
- 31 people
- Sponsor
- Catabasis Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Congenital Myopathy, Neuromuscular Diseases
Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment
Congenital myopathies (CM) is a large group of muscle disorders, presenting with hypotonia and non-progressive generalised muscle weakness, which can lead to motor developmental delay.More than 20 genes can cause CM and currently…
- Conditions
- Congenital Myopathy, Neuromuscular Diseases, Musculoskeletal Diseases, Nemaline Myopathy
- Trial name
- COMPIS (NCT05099107)
- Testing
- Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet, Salbutamol Only Product in Oral Dose Form
- Where
- Sweden
- Volunteers
- 18 people
- Sponsor
- Vastra Gotaland Region
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
Limb-girdle Muscular Dystrophy
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy…
- Trial name
- NCT04475926
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 205 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
Objective: The objective of this observational study is to evaluate and quantify trunk muscle compensatory movement patterns in patients with Duchenne Muscular Dystrophy (DMD) using computer vision technology.
- Trial name
- NCT06755138
- Testing
- Observational Assessment
- Where
- South Korea
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Seoul National University Hospital
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.