Clinical trials: Severe Muscle Atrophy
Duchenne or Severe Becker Myopathy
IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère
Clinical, prospective and monocentric study aiming at assessing the feasibility of fibrosis detection and quantification (and of function assessment) during MRI without breath-holds in a population of adults and children with…
- Trial name
- NCT02078076
- Testing
- Magnetic Resonance Cardiac Imaging (with Gadolinium)
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Sarcopenia, COPD
Evaluate the Effect of Dietary Supplement Combined Exercise Among Patients With Sarcopenia Comorbid With Lung Disease
This trial is a prospective multi-center study. The purpose of this study is to evaluate the effect of dietary supplement combined with supervised exercised on the physical performance, body composition and lung function among…
- Conditions
- Sarcopenia, COPD, ILD
- Trial name
- NCT05926713
- Testing
- Sarcojoint®, supervised exercise
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- Orient Europharma Co., Ltd.
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
Spinal Muscular Atrophy, Muscular Dystrophy
National Registry for Egyptian Pediatric Neuromuscular Diseases
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes
- Trial name
- NCT02124616
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Ain Shams University
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle…
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
- Trial name
- CMDPROS (NCT01403402)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Cure CMD
Cardiomyopathies, Amyloidosis Cardiac
Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
The study aims to identify new diagnostic and prognostic markers for CMP that can help predict disease progression. In particular, the study will focus on microRNAs (miRNAs) and spatial transcriptomics, which are emerging…
- Conditions
- Cardiomyopathies, Amyloidosis Cardiac, Fabry Disease, Arrhythmogenic Cardiomyopathy
- Trial name
- CMPMIRNA (NCT06813443)
- Where
- 3 sites in Italy
- Volunteers
- 700 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Cerebral Palsy, Arthrogryposis
Effects of Power Mobility on Young Children With Severe Motor Impairments
The purpose this study is to determine the effects of power mobility on the development and function of young children of young children whose severe physical disabilities limit their exploratory behaviors and may unnecessarily…
- Conditions
- Cerebral Palsy, Arthrogryposis, Spinal Muscular Atrophy, Other Central Nervous System or Musculoskeletal Disorders
- Trial name
- NCT01028833
- Testing
- Power mobility
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Oklahoma
Severe Muscle Atrophy, Muscle Weakness
BFRT for Severe Lower Extremity Muscle Atrophy
Muscle weakness or atrophy is a common condition following acute and chronic musculoskeletal injuries. Strength training is an imperative component in clinical rehabilitation of musculoskeletal injuries.
- Trial name
- BFRT (NCT04357184)
- Testing
- Blood flow restriction training
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Cincinnati Sportsmedicine Research and Education Foundation
Osteoporosis, Muscular Dystrophy
Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy
By supplying an adequate amount of calcium and vitamin D with the addition of weekly bisphosphonate, the investigators will be able to increase bone mass and decrease the incidence of fragility fractures in these children with…
- Conditions
- Osteoporosis, Muscular Dystrophy, Cystic Fibrosis
- Trial name
- NCT01882400
- Testing
- Bisphosphonate treatment
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Gilles Boire
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Stroke, Osteoporosis
Sarcopenia and Risk of Falls in Patients With Major Chronic Diseases
The objective of this study is to investigate the effects of therapeutic exercise and nutrition intervention for sarcopenia and risk of falls in patients with major chronic diseases.
- Conditions
- Stroke, Osteoporosis, Chronic Kidney Diseases, Cancer
- Trial name
- NCT03798418
- Testing
- elastic band strengthening exercise, diet counseling
- Where
- Taiwan
- Volunteers
- 186 people
- Sponsor
- Changhua Christian Hospital