Clinical trials: Muscle Disorders
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Biomarker Development in LGMD2i
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD2i to support therapeutic development.
- Trial name
- MLB-01-001 (NCT04202627)
- Where
- 11 sites in United States, Denmark
- Volunteers
- 101 people
- Sponsor
- ML Bio Solutions, Inc.
Spinal Muscular Atrophy
Implementation of FEES in Spinal Muscle Atrophy
The major aim of this project is to assess comprehensively frequency and extent of dysphagia and bulbar dysfunction in SMA1, 2, and 3 patients by applying FEES and validated dysphagia scores.
- Trial name
- DYS-SMA (NCT04773470)
- Testing
- Flexible Endoscopic Evaluation of Swallowing
- Where
- Germany
- Volunteers
- 79 people
- Sponsor
- University of Giessen
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Peritoneal Neoplasms, Retroperitoneal Neoplasms
Continuous Hyperthermic Peritoneal Perfusion (CHPP) With Cisplatin for Children With Peritoneal Cancer
There has been no successful treatment of diffuse peritoneal metastasis or carcinomatosis, in childhood tumors. Once this advanced stage of disease is evident, survival is measured in weeks.
- Conditions
- Peritoneal Neoplasms, Retroperitoneal Neoplasms, Gastrointestinal Neoplasms, Adenocarcinoma
- Trial name
- NCT00436657
- Testing
- CHPP of Cisplatin, Abdominal Surgery
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- M.D. Anderson Cancer Center
Fibroid, Miscarriage in First Trimester
Fibroids and the Risk of Miscarriage: a Prospective Observational Study in an Early Pregnancy Population
The aim of this observational study is to whether fibroids increase the risk of miscarriage in women in our early pregnancy population.
- Conditions
- Fibroid, Miscarriage in First Trimester, Miscarriage in Second Trimester
- Trial name
- FiRM (NCT06986616)
- Where
- United Kingdom
- Volunteers
- 436 people, healthy volunteers welcome
- Sponsor
- King's College Hospital NHS Trust
GNE Myopathy, GNE Related Diseases
A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
Background: GNE Myopathy is a disease that causes walking difficulties and increasing muscle weakness. It usually develops in young adults (between 20 and 30 years of age), and affects arm and leg muscles.
- Trial name
- NCT01417533
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Strabismus, Esotropia
Bupivacaine Injection of Eye Muscles to Treat Strabismus
This study seeks to determine if bupivacaine injection of eye muscles can make them stronger and stiffer, and thereby correct the position of eyes that are turned in or mis-aligned, a condition generally termed strabismus.
- Conditions
- Strabismus, Esotropia, Exotropia, Graves Disease
- Trial name
- NCT01616108
- Testing
- Bupivacaine
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Smith-Kettlewell Eye Research Institute
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
- Trial name
- LELANTOS-2 (NCT04632940)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 52 sites in United States, Australia and 9 more
- Volunteers
- 73 people
- Sponsor
- Kyntra Bio
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT02052791
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 47 people
- Sponsor
- Biogen
Gulf War Illness, Persian Gulf War Syndrome
Study of Gulf War Illness (GWI) by Comparing GWI and Healthy Veterans
Determine if: genetic differences of CNDP1 gene the previously defined GWI/Chronic Fatigue Syndrome (GWI/CFS) cerebrospinal fluid proteome contents psychometric other variables can differentiate between veterans of the 1990-1991…
- Trial name
- NCT00810225
- Where
- United States
- Volunteers
- 516 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals
Temporomandibular Joint Disorders
Evaluation of C-reactive Protein Level in the Group of Patient With Pain Form of Temporomandibular Joint Dysfunction
Temporomandibular joints dysfunction is a functional disorder concern with the abnormal functioning of the muscles of the stomatognathic system, temporomandibular joints involved in the dynamic movements of the jaw and…
- Trial name
- NCT03065608
- Volunteers
- 72 people
- Sponsor
- Jagiellonian University
Interstitial Lung Disease, Skeletal Muscle Dysfunction
Assessment of Skeletal Muscle Dysfunction in Patient With Idiopathic Pulmonary Fibrosis
Interstitial lung disease includes a heterogeneous group of chronic lung conditions that is characterized by exertional dyspnoea and poor health related quality of life .
- Trial name
- NCT03440489
- Testing
- Six-minute walking test, 30 seconds chair stand test
- Volunteers
- 1 people
- Sponsor
- Assiut University
Blood Pressure, Depression
Essential Hypotension and Allostasis Registry
The essential arterial hypotension and allostasis registry is a prospective, observational research that has the purpose of demonstrating that essential blood pressure (BP) disorders and the associated comorbidities are a result…
- Conditions
- Blood Pressure, Depression, Panic Attack, Fibromyalgia
- Trial name
- ESSENTIAL (NCT02018497)
- Where
- Colombia
- Volunteers
- 5,000 people
- Sponsor
- CES University
Blepharoptosis, Ptosis, Eyelid
Clinical Trial Comparing Two Non-Surgical Treatments for Severe Blepharoptosis
This clinical trial will evaluate two non-surgical devices designed to improve eye lid opening for patients with severe Blepharoptosis (incomplete opening of the eyelids).
- Conditions
- Blepharoptosis, Ptosis, Eyelid, Myasthenia Gravis, Stroke
- Trial name
- NCT04678115
- Testing
- Magnetic Levator Prosthesis (MLP), Kinesiotape Frontalis Sling (KTFS)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Duchenne Muscular Dystrophy (DMD)
The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD
Background: Duchenne muscular dystrophy (DMD) is an X chromosome recessive hereditary disease and mainly characterized by progressive muscle weakness and atrophy.
- Trial name
- NCT02614820
- Testing
- Remote Ischemic Preconditioning Training Apparatus
- Where
- China
- Volunteers
- 100 people
- Sponsor
- General Hospital of Chinese Armed Police Forces
Spinal Curvatures
Effect of Sugammadex for Muscle Motor Response and Awareness in Intraoperative Wakeup
The study will be performed to evaluate motor response and awareness with using sugammadex during wakeup procedure in spine surgery.
- Trial name
- NCT02390817
- Testing
- Sugammadex, Neostigmine
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Cukurova University
Eosinophilia-Myalgia Syndrome
L-5-HTP-Related EMS
In 1989 more than 1500 people who took the dietary supplement L-tryptophan for insomnia and depression developed eosinophilia myalgia syndrome (EMS)-a potentially fatal disease characterized by an excess of a type of white blood…
- Trial name
- NCT00001918
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Mental Health (NIMH)
Stroke
Effects of Electromyography-Triggered Constraint-Induced Movement Cycling Therapy on Patients With Chronic Stroke
The study investigates the potential of Electromyography (EMG)-triggered Constraint-Induced Movement Cycling Therapy (CIMCT) versus General Cycling Training (GCT) to enhance balance, strength, and daily activities in chronic…
- Trial name
- NCT06367140
- Testing
- EMG-triggered CIMCT device, general cycling training
- Where
- South Korea
- Volunteers
- 58 people
- Sponsor
- Sahmyook University
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