Clinical trials: DMD
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Urinary Titin Biomarker in DMD
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful…
- Trial name
- NCT07332013
- Testing
- Descending stair walk
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS)
Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias
Human induced pluripotent stem cells (hiPSCs) have driven a paradigm shift in the modeling of human disease; the ability to reprogram patient-specific cells holds the promise of an enhanced understanding of disease mechanisms and…
- Conditions
- Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
- Trial name
- NCT02413450
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Develop a New Group Version of the Becker-DeGroot-Marsckek (BDM) Auction to Measure Willingness to Pay of Compound Members for Shared Hardware., Develop a New Survey Instrument to Measure Behavioural Determinants of Hand Washing and Water Treatment Like Disgust and Shame or Social Pressure.
Disgust and Shame Based Safe Water and Handwashing Promotion
An estimated 2.2 million children under the age of 5 years die from diarrheal disease each year. Most of the burden of diarrheal disease is thought to be preventable with improvements in sanitation, water quality, and hygiene.
- Conditions
- Develop a New Group Version of the Becker-DeGroot-Marsckek (BDM) Auction to Measure Willingness to Pay of Compound Members for Shared Hardware., Develop a New Survey Instrument to Measure Behavioural Determinants of Hand Washing and Water Treatment Like Disgust and Shame or Social Pressure., Identify New Methods for Measuring Hand Washing and Water Treatment Behaviour., Compare the Effectiveness of the Disgust and Shame Based Interventions With Standard Public Health Interventions.
- Trial name
- NCT02037243
- Testing
- Standard Public Health Intervention with Water Treatment, Standard Public Health Intervention with Water Treatment & Hand Washing
- Where
- Bangladesh
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- International Centre for Diarrhoeal Disease Research, Bangladesh
Duchenne Muscular Dystrophy (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Duchenne Muscular Dystrophy
Dual Task in Duchenne Muscular Dystrophy
This study was planned to determine the effects of the dual-task performance of children with DMD with motor dysfunction and varying degrees of cognitive impairment compared to their healthy peers, to compare the dual-task…
- Trial name
- NCT05575648
- Testing
- 10 meter walk test
- Where
- Turkey (Türkiye)
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Muscular Diseases
Non-invasive Molecular Imaging of Muscle Structure (MSOT_muscles)
This study aims to determine hemoglobin and collagen levels in muscles before and after exercise, and over time, using Multispectral Optoacoustic Tomography (MSOT).
- Trial name
- MSOT_muscles (NCT03979157)
- Testing
- Multispectral Optoacoustic Tomography
- Where
- Germany
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Duchenne Muscular Dystrophy, Burden, Dependency
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
The purpose of this study is to improve the understanding of the treatment goals that a person with Duchenne Muscular Dystrophy (DMD) or the caregiver may be most interested in, based on the severity of the person's disease.
- Conditions
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management
- Trial name
- NCT03680365
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Jett Foundation, Inc.
Duchenne Muscular Dystrophy
The Burden of Access in Duchenne Muscular Dystrophy in the US
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these…
- Trial name
- NCT03951675
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Florida
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Duchenne Muscular Dystrophy
Evaluation of Home Based Assessments on Participants With DMD
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
- Trial name
- NCT05657938
- Testing
- Wearable Device, Duchenne Video Assessment (DVA)
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Solid Biosciences Inc.
Becker Muscular Dystrophy
Tadalafil in Becker Muscular Dystrophy
Summary for Patients: This study, funded by the Muscular Dystrophy Association, is intended to build on recent findings published in the journal Nature showing beneficial effects of tadalafil (also known as Cialis) in mice with…
- Trial name
- NCT01070511
- Testing
- Tadalafil
- Where
- 2 sites in United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy
The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy
Children with Duchenne Muscular Dystrophy (DMD) have difficulties towards the end of the ambulatory period, especially in activities that require lower extremity proximal muscle strength such as walking, climbing stairs, standing…
- Trial name
- NCT04287582
- Testing
- Electromyographic device
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency)
Evaluation of Limb-Girdle Muscular Dystrophy
The purpose of this study is to understand the biochemistry of different types of Limb-Girdle Muscular Dystrophy (LGMD) and to determine appropriate outcome measures for future clinical treatment trials for LGMD.
- Conditions
- Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
- Trial name
- NCT00893334
- Where
- 2 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy (DMD)
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.
- Trial name
- IBISD (NCT01380964)
- Where
- France
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- Genethon
Becker Muscular Dystrophy
Sodium Nitrate to Improve Blood Flow
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD).
- Trial name
- NCT02847975
- Testing
- Sodium nitrate
- Where
- 2 sites in United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI)
Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
The primary aim of this observational study is to investigate specific aspects of social cognition in dystrophinopathies. Body awareness, interpersonal distance and emotional processing will be measured in a sample of patients…
- Conditions
- Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI), Social Cognition
- Trial name
- NCT06874166
- Testing
- Electroencephalography (EEG), Social Cognition Tasks
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Muscular Dystrophy, Duchenne Muscular Dystrophy,
Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy.
- Trial name
- mdp (NCT01834066)
- Testing
- Stem Cell
- Where
- India
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Chaitanya Hospital, Pune
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