Clinical trials: Congenital Myopathy
Duchenne's Muscular Dystrophy
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD).
- Trial name
- NCT02235844
- Testing
- Umbilical Cord Mesenchymal Stem Cells
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Allergy and Asthma Consultants, Wichita, Kansas
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Electromyography, EMG: Myopathy
Electromyographic Study for the Help and Guidance of BoNTA Administration in the Treatment of Chronic Pelvic Floor Pain
Chronic pelvic pain (CPP) is a common presenting complaint affecting approximately 15-40 % of women aged 18-50 in western countries and 5-43% of women in most developing countries.
- Conditions
- Electromyography, EMG: Myopathy, EMG Syndrome, Pelvic Pain
- Trial name
- SEMG (NCT03715777)
- Testing
- Clostridium botulinum type A (BoNTA)
- Where
- Spain
- Volunteers
- 25 people
- Sponsor
- Jose Alberola-Rubio
Cerebral Palsy, Spina Bifida
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Muscular Dystrophies, Musculoskeletal Disorder
- Trial name
- NCT03851107
- Testing
- Engagement in 6-week community-based activity program
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- McGill University
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
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
The task consists in reach as much bubbles as they can, the bubbles appear on the screen of the computer and should be reached in 10 seconds.
- Trial name
- NCT02891434
- Testing
- Acquisition on TouchScreen, Acquisition on Kinect, Acquisition on LeapMotion
- Where
- Brazil
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Facioscapulohumeral
Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1).
- Trial name
- NCT06131983
- Testing
- SRP-1001 for Injection
- Where
- 16 sites in Australia, Canada and 5 more
- Volunteers
- 60 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in…
- Trial name
- NCT02834650
- Testing
- Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test
- Where
- 3 sites in United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Stanford University
Duchenne Muscular Dystrophy
Exploratory Study of NS-065/NCNP-01 in DMD
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT02081625
- Testing
- NS-065/NCNP-01
- Where
- Japan
- Volunteers
- 10 people
- Sponsor
- National Center of Neurology and Psychiatry, Japan
Laminopathies, Emerinopathies
Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)
Laminopathies and emerinopathies are complex group of rare disorders due to mutations in A-type lamins (LMNA) and Emerin (EMD) genes.
- Trial name
- OPALE (NCT03058185)
- Where
- 29 sites in France
- Volunteers
- 800 people
- Sponsor
- Pitié-Salpêtrière Hospital
Duchenne Muscular Dystrophy
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
- Trial name
- NCT06328725
- Testing
- EN001
- Where
- 2 sites in South Korea
- Volunteers
- 88 people
- Sponsor
- ENCell
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases.
- Conditions
- Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Congenital Myopathy
- Trial name
- ARNseq-Musc (NCT06833489)
- Testing
- ARN extraction from muscle biopsies
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2I
Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I
The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
- Trial name
- NCT02165358
- Where
- Denmark
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy, Muscle Strength
Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy
Investigator researched that the effect of trunk and lower extremity muscle strength on hamstring flexibility in children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Muscle Strength, Lower Extremity, Hamstring Contractures
- Trial name
- NCT03589573
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Limb Girdle Muscular Dystrophy
Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts
Limb girdle muscular dystrophies were originally defined as a postnatal progressive muscle disease, which begins and primarily affects the pelvic and scapular muscles.
- Trial name
- CALNATHIS (NCT06390566)
- Testing
- calpaïnopathies
- Where
- France
- Volunteers
- 25 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Molar Incisor Hypomineralization
The Effect of Molar Incisor Hypomineralisation on the Stomatognathic System
This study aims to examine whether children with Molar Incisor Hypomineralization (MIH) are more likely to have problems with the jaw joint and chewing muscles.
- Trial name
- NCT06951139
- Testing
- Ultrasound and Shear Wave Elastography
- Where
- Turkey (Türkiye)
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Ozge Gungor
Muscular Dystrophy, Duchenne
Safety Study of BLS-M22 in Healthy Volunteers
BLS-M22 is being developed as an anti-myostatin agent for the treatment of Duchenne Muscular Dystrophy (Muscular Dystrophy). A total of 37 subjects participated in this study to confirm the safety of BLS-M22.
- Trial name
- NCT03789734
- Testing
- BLS-M22
- Where
- South Korea
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- BioLeaders Corporation
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
Myotonic Dystrophy
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.
This is an open-label extension study intended to evaluate the long-term safety and efficacy of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2) who have completed the parent study MEX-DM-302.
- Trial name
- NCT06549400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 6 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.
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