Clinical trials: Muscle Disorders
Muscular Dystrophy, Duchenne
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping…
- Trial name
- NCT03375255
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy, Upper Extremity Problem
Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05274555
- Testing
- Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 41 people
- Sponsor
- Dokuz Eylul University
Duchenne Muscular Dystrophy, Natural History
phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function
- Conditions
- Duchenne Muscular Dystrophy, Natural History, Motor Function; Retardation
- Trial name
- GUP21003 (NCT06366815)
- Testing
- Different Phenotypes of non ambulant patients with Duchenne Muscular Dystrophy
- Where
- 2 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Duchenne Muscular Dystrophy
Stacking Exercises Aid the Decline in FVC and Sick Time
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths…
- Trial name
- STEADFAST (NCT01999075)
- Testing
- Lung Volume Recruitment (LVR), Conventional Treatment
- Where
- 9 sites in Canada
- Volunteers
- 70 people
- Sponsor
- Children's Hospital of Eastern Ontario
Pediatric Obesity
Inspiratory Muscle Rehabilitation in Children With Obesity
This is a single-center, randomized, SHAM-controlled, parallel assignment, double-masked, interventional trial among children aged 8-17 years (not yet 18 years old) of age with obesity (n=30), recruited from the Duke Healthy…
- Trial name
- BREATHE Fit (NCT05412134)
- Testing
- Pro2 - 75% of participant's MIP, Pro2 - 15% of participant's MIP
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Duke University
Muscular Dystrophies
The Efficacy of Using Far Infrared Radiation to Manage Muscular Dystrophies
The muscular dystrophies (MD) are a group of more than 30 neuromuscular disorders that are characterized by progressive skeletal muscle weakness, defects in muscle proteins and the death of muscle cells and tissue.
- Trial name
- NCT00674843
- Testing
- Far Infrared Radiation
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- GAAD Medical Research Institute Inc.
Spinal Muscular Atrophy
Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System
In this project, we will establish the efficient and accurate gene dose determination system by combining the heterodulex analysis and gene dose analysis on DHPLC platform based on various quantitative and multiplex PCR…
- Trial name
- NCT00155168
- Where
- Taiwan
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Retinoblastoma, Clear Cell Sarcoma
Simvastatin With Topotecan and Cyclophosphamide in Relapsed and/or Refractory Pediatric Solid and CNS Tumors
This is a Phase I trial with new experimental drugs such as simvastatin in combination with topotecan and cyclophosphamide in the hopes of finding a drug that may work against tumors that have come back or that have not responded…
- Conditions
- Retinoblastoma, Clear Cell Sarcoma, Renal Cell Carcinoma, Rhabdoid Tumor
- Trial name
- AflacST1402 (NCT02390843)
- Testing
- Simvastatin, Cyclophosphamide, Topotecan
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Emory University
Mitochondrial Myopathies
NiaMIT Continuation With Early-stage Mitochondrial Myopathy Patients
The most frequent form of adult-onset mitochondrial disorders is mitochondrial myopathy, often manifesting with progressive external ophthalmoplegia (PEO), progressive muscle weakness and exercise intolerance.
- Trial name
- NCT04538521
- Testing
- Niacin
- Where
- Finland
- Volunteers
- 3 people
- Sponsor
- University of Helsinki
Hypotonia
Osteopathic Treatment on Motor Development of Hypotonic Infants.
Interventional study involving the human at risk and minimal constraint (RIPH2), randomized single-blind comparing the impact of osteopathic treatment on the axial tone of the hypotonic infant versus simulation
- Trial name
- HYPOSTEO (NCT03852004)
- Testing
- ostheopathy, simulated osteopathic treatment
- Where
- 2 sites in France
- Volunteers
- 40 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Nemaline Myopathy, Myopathies
Natural History Study for Patients With Nemaline Myopathy in Spain
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of nemaline myopathies.
- Conditions
- Nemaline Myopathy, Myopathies, Myopathic Conditions, NEB
- Trial name
- NCT07488806
- Testing
- Muscle Ultrasound, Motor function scales, Complete physical examination
- Where
- Spain
- Volunteers
- 100 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Muscular Atrophy, Spinal
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
- Trial name
- NCT02240355
- Testing
- RO6885247
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 9 people
- Sponsor
- Hoffmann-La Roche
Cerebral Palsy
The Effect of Treadmill Back Walking Training on Balance and Function in Cerebral Palsy
The aim of this study was to investigate the effect of treadmill back walking training on balance and function in cerebral palsy.
- Trial name
- CP (NCT04136678)
- Testing
- walking training
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Istanbul Medipol University Hospital
Temporomandibular Joint Disorders
Stimulation of the Proprioceptive Trigeminocardiac Reflex
Study over muscular stretching to reduce pain the oral cavity.
- Trial name
- PTCRSTIM (NCT02908568)
- Testing
- Spring device, Mandible stimulator
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Azienda USL 12 Versilia
Pes Planus
Foot Muscle Strengthening Exercises for Pes Planovalgus
Pes planovalgus is a common foot condition affecting the child population. It is characterized by the partial or complete collapse of the medial longitudinal arch with rearfoot eversion and forefoot abduction, which is associated…
- Trial name
- NCT06548750
- Testing
- Intrinsic and Ectrinsic Exercise, Intrinsic Exercise
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Istanbul University - Cerrahpasa
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)
Cerebral Palsy
Plyometric Exercise-induced Mechano-morphological Changes of Plantar Flexor Muscle-tendon Unit in Cerebral Palsy
This study was designed to investigate the effect of three months of plyometric exercise training on the mechanical and morphological properties of the plantar flexor muscle-tendon unit in children with unilateral cerebral palsy…
- Trial name
- NCT05301738
- Testing
- Plyometric Exercises, Standard Physical Therapy
- Where
- Saudi Arabia
- Volunteers
- 38 people
- Sponsor
- Cairo University
Congenital Myotonic Dystrophy
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
This treatment plan is limited to a single patient with Congenital Myotonic Dystrophy, who is ineligible or otherwise unable to participate in ongoing clinical trials.
- Trial name
- NCT07119775
- Testing
- Tideglusib
- Sponsor
- AMO Pharma Limited
Choreatic Disorder
Heart and Skeletal Muscle Problems in Neuroacanthocytosis
The purpose of this study is to learn about heart and skeletal muscle problems related to neuroacanthocytosis (also called Mcleod's syndrome and Levine-Critchley disease).
- Trial name
- NCT00007228
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchene Muscular Dystrophy
Home-Based Tele-Rehabilitation for Respiratory Function in Children With Duchenne Muscular Dystrophy
This study evaluates the feasibility and preliminary clinical effects of a home-based tele-rehabilitation program aimed at improving respiratory function in children with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT07629284
- Testing
- Home-Based Respiratory Telerehabilitation Program
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Samsung Medical Center
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.