Clinical trials: Muscle Disorders
Duchenne Muscular Dystrophy
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
There isn't specific Health related quality of life measure for children with DMD in French. The aim of this study is to validate the French version of the Pediatric Quality of Life Inventory 3.0 Duchenne Muscular Dystrophy…
- Trial name
- ValPedsQLDMD (NCT03513367)
- Testing
- Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale, The following data of motor function
- Where
- France
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Osteosarcoma, Ewing Sarcoma
Neoadjuvant Dual Checkpoint Inhibition and Cryoablation in Relapsed/Refractory Pediatric Solid Tumors
The is a phase II, single arm, open-label, multi-site trial studying the combination of cryoablation therapy and dual checkpoint inhibition with nivolumab (anti-PD-1) and ipilimumab (anti-CTLA-4) given at the recommended phase 2…
- Conditions
- Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Relapsed Pediatric Solid Tumor
- Trial name
- NCT05302921
- Testing
- Cryoablation Therapy, Nivolumab, Ipilimumab
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's National Research Institute
Spinal Muscular Atrophy
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal…
- Trial name
- NCT02644668
- Testing
- Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Where
- 19 sites in United States, Canada
- Volunteers
- 70 people
- Sponsor
- Cytokinetics
Neuromuscular Disorders, Neurodevelopmental Disorders
Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.
Neurodevelopmental disorders often result in abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as the inability to stand and walk.
- Trial name
- NCT07159360
- Testing
- CLINICAL EXPLORER
- Where
- 2 sites in Mexico, Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- MarsiBionics
Intraabdominal Hypertension
an Educational Program About Abdominal Compartment Syndrome
Early detection of intra-abdominal hypertension is essential to the prevention of abdominal compartment syndrome and requires close surveillance of intra-abdominal hypertension in patients at increased risk.
- Trial name
- Ghada (NCT02639013)
- Testing
- transducer technique
- Where
- Egypt
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Assiut University
Cystic Fibrosis (CF), Cystic Fibrosis in Children
Physical Impairments in Children With Cystic Fibrosis
It is aimed to reveal impairments regarding urinary incontinence, dyspnea, muscle strength, functional capacity or quality of life in children and adolescents with cystic fibrosis compared to healthy children and adolescents.
- Trial name
- NCT06984679
- Testing
- Physical Evaluations in Children with Cystic Fibrosis, Physical Evaluations in Healthy Children
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people
- Sponsor
- Izmir Democracy University
Vegetative State, Minimally Conscious State
Multichannel tDCS to Reduce Hypertonia in Patients With Prolonged DOC
Previous studies showed that transcranial direct current stimulation (tDCS) transiently improves performance of motor function in stroke patients, as well as decrease muscle hypertonia.
- Conditions
- Vegetative State, Minimally Conscious State, Spasticity, Muscle, Disorder of Consciousness
- Trial name
- NCT03797573
- Testing
- tDCS
- Where
- Belgium
- Volunteers
- 17 people
- Sponsor
- University of Liege
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
Ulcerative Colitis
Effects of WB-EMS in Ulcerative Colitis Patients
The purpose of this study is to determine the effect of 12 weeks of WB-EMS either applicated passive in a supine resting position or active during slight movements compared with a non-training control group on body composition…
- Trial name
- NCT02144129
- Testing
- active Whole Body Electromyostimulation, Passive WB-EMS, WB-EMS application
- Where
- Germany
- Sponsor
- University of Erlangen-Nürnberg Medical School
Facio-Scapulo-Humeral Dystrophy
FSHD Molecular Characterization
To characterize the clinical and molecular phenotype of FSHD.
- Trial name
- NCT06096441
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Nationwide Children's Hospital
Spinal Muscular Atrophy
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S.
- Trial name
- WeSMA (NCT05232929)
- Testing
- Risdiplam
- Where
- 38 sites in United States, Puerto Rico
- Volunteers
- 403 people
- Sponsor
- Genentech, Inc.
Spinal Muscular Atrophy
Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
The goal of this clinical trial is to study respiratory muscle training in patients with Spinal Muscular Atrophy (SMA). The main questions it aims to answer are: Is a home-based individualized training program for the inspiratory…
- Trial name
- RESISTANT (NCT05632666)
- Testing
- Inspiratory and expiratory muscle training at a therapeutic intensity, Inspiratory and expiratory muscle training at a non-therapeutic intensity
- Where
- Netherlands
- Volunteers
- 30 people
- Sponsor
- UMC Utrecht
Heart and Blood Disease, Muscle, Bone and Cartilage Disease
IRF-PAI Functional Outcomes Data(With FIM Instrument Variables)
IRF-PAI Functional Outcomes Data, including FIM instrument variables
- Conditions
- Heart and Blood Disease, Muscle, Bone and Cartilage Disease, Nervous System Diseases, Skin and Connective Tissue Disease
- Trial name
- NCT01779037
- Where
- United States
- Sponsor
- Uniform Data System for Medical Rehabilitation
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Ewing's Sarcoma, Leukemia
Evaluation, Treatment, and Natural History of Children and Young Adults With Cancer or Rare Diseases
This protocol is designed to evaluate children with cancer who appear to be probable candidates for future protocol entry or have disease manifestations that are of unique scientific importance or educational value.
- Conditions
- Ewing's Sarcoma, Leukemia, Lymphoma, Brain Tumors
- Trial name
- NCT00001686
- Where
- United States
- Volunteers
- 482 people
- Sponsor
- National Cancer Institute (NCI)
Duchenne Muscular Dystrophy
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S.
- Trial name
- NCT02592941
- Testing
- Deflazacort
- Where
- 42 sites in United States
- Sponsor
- PTC Therapeutics
Cancer
Antithymocyte Globulin, Clofarabine, and Rituximab in Treating Patients After an Unsuccessful Stem Cell Transplant
RATIONALE: Antithymocyte globulin, clofarabine, and rituximab may stop the patient's immune system from rejecting the donor's stem cells when they do not exactly match the patient's blood.
- Trial name
- NCT00617929
- Testing
- anti-thymocyte globulin, rituximab, clofarabine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Arthrogryposis Multiplex Congenita, AMC-Arthrogryposis Multiplex Congenita
Arthrogryposis Multiplex Congenita in Pediatric Age: Correlation Between MUScular MRI and Functional Evaluation
The aim of our study is to evaluate the correlation between quantified fibro-adipous infiltration of muscles on MRI assessed by MERCURI Score and the functional abilities (deficiencies, activity limitations and social…
- Conditions
- Arthrogryposis Multiplex Congenita, AMC-Arthrogryposis Multiplex Congenita, Pediatric ALL
- Trial name
- AMUSE (NCT05393375)
- Testing
- No intervention
- Where
- France
- Volunteers
- 97 people
- Sponsor
- University Hospital, Grenoble
Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis
This study will use a magnetic resonance imaging technique called nuclear magnetic spectroscopy (H-MRS) to define the pathology and progression of primary lateral sclerosis, hereditary spastic paraplegia and amyotrophic lateral…
- Conditions
- Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Amyotrophic Lateral Sclerosis
- Trial name
- NCT00023075
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Adult Alveolar Soft-part Sarcoma, Adult Angiosarcoma
Observation, Radiation Therapy, Combination Chemotherapy, and/or Surgery in Treating Young Patients With Soft Tissue Sarcoma
This phase III trial is studying observation to see how well a risk based treatment strategy works in patients with soft tissue sarcoma.
- Conditions
- Adult Alveolar Soft-part Sarcoma, Adult Angiosarcoma, Adult Epithelioid Sarcoma, Adult Extraskeletal Chondrosarcoma
- Trial name
- NCT00346164
- Testing
- doxorubicin hydrochloride, clinical observation, therapeutic conventional surgery
- Where
- 187 sites in United States, Australia and 3 more
- Volunteers
- 588 people
- Sponsor
- Children's Oncology Group
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