Clinical trials: Muscle Disorders
Polymyositis
Epidemiological Study of Inflammatory Myopathies in a French Region
Knowledge of the descriptive epidemiology of MIs is very limited. The main objective of this study is to determine the prevalence of inflammatory myopathies in Alsace at the 1st January 2012 with "capture-recapture" design using…
- Trial name
- PREMIA (NCT02881450)
- Volunteers
- 117 people
- Sponsor
- University Hospital, Strasbourg, France
Muscular Dystrophies, Congenital Myopathy
Bullying in Youth With Muscular Dystrophy and Congenital Myopathies
Bullying is an epidemic in Canada, and rates may be underreported. Youth with a disability were more likely to be bullied that those without disabilities, specifically if the disability was visible.
- Trial name
- NCT04733976
- Where
- 2 sites in Canada
- Volunteers
- 29 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Sarcopenia, Pediatric Cancer
Determination of Sarcopenia Risk and Related Factors in Pediatric Oncological Patients
In children, both malnutrition and sarcopenia are associated with prolongation of hospital stay, increased morbidity, mortality, and health-related complications.
- Trial name
- NCT05294380
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Trakya University
Duchenne Muscular Dystrophy
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD).
- Trial name
- IGNITE DMD (NCT03368742)
- Testing
- SGT-001
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Solid Biosciences Inc.
Polyneuropathy, Charcot Marie Tooth Disease (CMT)
Effects of Resistance Exercises in Hereditary Sensory-Motor Neuropathy (Charcot-Marie-Tooth Disease)
The goal of this clinical trial is to compare the effects of an 8-week resistance exercise training program on skeletal muscle quality, functional capacity, and quality of life in young individuals aged 15 to 29 years, with and…
- Trial name
- PNP (NCT07152197)
- Testing
- Progressive Resistance Exercise Training
- Where
- 2 sites in Chile
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Universidad de La Frontera
Urinary Incontinence
Whole Body Vibration and Pelvic Floor Exercises on Urinary Incontinence
Stress urinary incontinence is common in men following prostate cancer surgery. Rehabilitative interventions incorporate pelvic floor muscle training, biofeedback, electrical stimulation, lifestyle changes, or a combination of…
- Trial name
- [PTREC] (NCT03325660)
- Testing
- whole body vibration
- Volunteers
- 61 people
- Sponsor
- Ahlia University
Duchenne Muscular Dystrophy
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD.
- Trial name
- NCT00758225
- Testing
- Idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Peripheral Facial Paralysis, Facial Palsy
Functional Recovery From Facial Paralysis
This is a controlled, single-blind (hybrid) clinical investigation that aims to analyze the process of recovery of muscle function in people with peripheral facial paralysis who receive kinesthetic treatment with…
- Conditions
- Peripheral Facial Paralysis, Facial Palsy, Synkineses
- Trial name
- NCT06532903
- Testing
- Selective muscle electrostimulation with exponential current, Facial muscle reeducation in front of the mirror
- Where
- Argentina
- Volunteers
- 50 people
- Sponsor
- Universidad Nacional del Nordeste, Argentina
Spinal Muscular Atrophy
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) is neurodegenerative disease of anterior horn cells of spinal cord and represents the second more frequent pathology in childhood.
- Trial name
- NCT00961103
- Where
- Italy
- Volunteers
- 80 people
- Sponsor
- Azienda USL Reggio Emilia - IRCCS
Adolescent Idiopathic Scoliosis
Is Respiratory Muscle Strength, Peripheral Muscle Strength and Postural Control Affected in Scoliosis?
The vertebral column is a structure that transfers the weight of the head and torso to the lower extremity, provides trunk movements and protects the spinal cord.A three dimensional deformity involving lateral flexion of the…
- Trial name
- NCT05833425
- Testing
- Respiratory Pressure Meter (MicroRPM)
- Where
- Turkey (Türkiye)
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Bezmialem Vakif University
Sarcoma, Soft Tissue Sarcoma
A Phase 2 Study of 9-ING-41Combined With Chemotherapy in Adolescents and Adults With Advanced Sarcomas
9-ING-41 in combination with gemcitabine and docetaxel will lead to sustained disease control and/or increase the rates of objective response in patients with unresectable or metastatic soft tissue and bone sarcomas.
- Conditions
- Sarcoma, Soft Tissue Sarcoma, Metastatic Sarcoma, Undifferentiated Pleomorphic Sarcoma
- Trial name
- NCT04906876
- Testing
- Gemcitabine, Docetaxel, 9-ING-41
- Where
- 2 sites in United States
- Sponsor
- Brown University
Stage IVB Adult Soft Tissue Sarcoma, Stage IIB Adult Soft Tissue Sarcoma
Isolated Limb Perfusion of Melphalan With or Without Tumor Necrosis Factor in Treating Patients With Soft Tissue Sarcoma of the Arm or Leg
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Infusing melphalan directly to the tumor may kill more tumor cells and cause less damage to healthy tissues.
- Conditions
- Stage IVB Adult Soft Tissue Sarcoma, Stage IIB Adult Soft Tissue Sarcoma, Stage IIC Adult Soft Tissue Sarcoma, Recurrent Adult Soft Tissue Sarcoma
- Trial name
- NCT00019968
- Testing
- melphalan, tumor necrosis factor
- Sponsor
- National Institutes of Health Clinical Center (CC)
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A), LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Characterization of the Natural History of LAMA2-RD and Identification of Novel Disease Biomarkers
The goal of this observational study is to learn about the natural history and multi-organ involvement of Laminin-Alpha-2-Related Dystrophy (LAMA2-RD) in pediatric and adult patients.
- Conditions
- LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A), LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\), Merosin Deficient CMD (Full or Partial), Merosin Deficient Congenital Muscular Dystrophy
- Trial name
- NCT07125040
- Testing
- Cardiac MRI
- Where
- Italy
- Volunteers
- 45 people
- Sponsor
- Università Vita-Salute San Raffaele
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) (ICD-10 G93.3)
Munich ME/CFS Cohort Study
The Munich cohort study MUC-CFS aims at the characterization and long-term follow-up of myalgic encephalitis/chronic fatigue syndrome (ME/CFS) in young people aged up to 25 years, as well as at the identification of…
- Trial name
- MUC-CFS (NCT06005246)
- Where
- Germany
- Volunteers
- 200 people
- Sponsor
- Technical University of Munich
Dystonia, Drug Induced Dystonia
Clinical Validation of DystoniaNet Deep Learning Platform for Diagnosis of Isolated Dystonia
This research involves retrospective and prospective studies for clinical validation of a DystoniaNet deep learning platform for the diagnosis of isolated dystonia.
- Conditions
- Dystonia, Drug Induced Dystonia, Parkinson Disease, Essential Tremor
- Trial name
- NCT05317390
- Testing
- DystoniaNet-based diagnosis of isolated dystonia
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Massachusetts Eye and Ear Infirmary
Limb Girdle Muscular Dystrophy
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Compartment Syndromes
Long Term Outcomes of Pediatric Compartment Syndrome
The goal of this study is to review the etiology, diagnostic criteria, complications and outcome of acute pediatric compartment syndrome identified at The Children's Hospital of Western Ontario (CHWO) .
- Trial name
- NCT03540394
- Where
- Canada
- Volunteers
- 100 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.
- Trial name
- NCT04632953
- Testing
- No Intervention
- Where
- 16 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
PolG, Primary Mitochondrial Myopathy
Prospective Natural History Study of POLG Disease
The PIONEER study is a prospective, natural history study dedicated to characterizing the clinical progression of POLG-related disorders.
- Trial name
- PIONEER (NCT07775872)
- Where
- 21 sites in United States, Australia and 7 more
- Volunteers
- 300 people
- Sponsor
- The POLG Foundation
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