Clinical trials: Congenital Myopathy
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old.
- Trial name
- NCT02760264
- Testing
- Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
Flat Feet, Flexible Flatfoot
Effect of Insole Added to Exercise in Patients With Bilateral Flexible Flatfoot
Pesplanus, commonly known as 'flatfoot,' is a common pathomechanical condition characterized by a lowered medial longitudinal arch (MLA) and exaggerated pronation.
- Conditions
- Flat Feet, Flexible Flatfoot, Balance; Distorted, Muscle Weakness
- Trial name
- NCT05056298
- Testing
- Custom made insole, Strengthening exercises of extrinsic foot muscles, Strengthening exercises of intrinsic foot muscles
- Where
- Egypt
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Horus University
Facioscapulohumeral Muscular Dystrophy, FSHD
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1
- Trial name
- FORTITUDE-3 (NCT07038200)
- Testing
- AOC-1020
- Where
- 46 sites in United States, Canada and 8 more
- Volunteers
- 200 people
- Sponsor
- Avidity Biosciences, Inc.
Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Limb Girdle Muscular Dystrophy (LGMD) Natural History
The purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD…
- Trial name
- NCT01783509
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Wake Forest University Health Sciences
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
Myotonic Dystrophy
Efficacy and Safety of DHEA for Myotonic Dystrophy
To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy
- Trial name
- NCT00167609
- Testing
- dehydroepiandrosterone 100 and 400 mg
- Where
- 10 sites in France
- Volunteers
- 75 people
- Sponsor
- University of Versailles
Muscular Dystrophies, Myopathies
Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
This study has the general objective of observing walking parameters during a clinical test to objectively estimate fatigue in patients with neuromuscular diseases.
- Conditions
- Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
- Trial name
- NCT06666816
- Testing
- inapplicable
- Where
- 2 sites in Italy
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Duchenne Muscular Dystrophy
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance…
- Trial name
- NCT01523964
- Testing
- Testing with EIM
- Where
- 6 sites in United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Dart Therapeutics. LLC
Limb-girdle Muscular Dystrophy
MRI and Muscle Involvement in Patients With Mutations in GMPPB
Limb girdle muscular dystrophies (LGMD) are a very heterogeneous group of muscle disorders characterized by muscle weakness and atrophy of the proximal muscles of the shoulder and pelvic girdles.
- Trial name
- NCT02635321
- Where
- Denmark
- Volunteers
- 4 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Becker Muscular Dystrophy
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.
- Trial name
- NCT01856868
- Testing
- (-)-epicatechin
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Craig McDonald, MD
Duchenne Muscular Dystrophy
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
The study is to demonstrate non-inferiority of spironolactone vs. eplerenone in preserving cardiac and pulmonary function in patients with preserved LV ejection fraction.
- Trial name
- NCT02354352
- Testing
- Eplerenone, Spironolactone
- Where
- 6 sites in United States
- Volunteers
- 52 people
- Sponsor
- Ohio State University
Myotonic Dystrophy Type 1 (DM1)
Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1
Myotonic Dystrophy type 1 (DM1) is a genetic multisystem disease causing muscle weakness and myotonia. As a result, upper limb function might become impaired.
- Trial name
- Technorehab (NCT05560438)
- Testing
- Tyromotion Amadeo and Armeo Senso
- Where
- Norway
- Volunteers
- 6 people
- Sponsor
- University of Oslo
Duchenne Muscular Dystrophy
Open-label Extension of the HOPE-2 Trial
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up.
- Trial name
- HOPE-2-OLE (NCT04428476)
- Testing
- Deramiocel (CAP-1002)
- Where
- 5 sites in United States
- Volunteers
- 13 people
- Sponsor
- Capricor Inc.
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy.
- Trial name
- NCT04281485
- Testing
- PF-06939926
- Where
- 53 sites in United States, Australia and 13 more
- Volunteers
- 114 people
- Sponsor
- Pfizer
Dysferlinopathy, LGMD2B Now Classified as LGMDR2
Clinical Outcome Study for Dysferlinopathy
The "Clinical Outcome Study for Dysferlinopathy" is being performed in centres in Europe (UK- Newcastle; Spain- Barcelona, Sevilla; San Sebastian;Denmark, Copenhagen, Italy- Padova; France- Paris,), USA (Charlotte, NC; Columbus…
- Conditions
- Dysferlinopathy, LGMD2B Now Classified as LGMDR2, Miyoshi Myopathy
- Trial name
- Jain COS (NCT01676077)
- Where
- 16 sites in United States, Chile and 7 more
- Volunteers
- 200 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Myotonic Dystrophy Type 1
Multicenter Observational Study of Myotonic Dystrophy Type 1
The purpose of the study is to determine the best ways to assess how people are affected by myotonic dystrophy type 1 (DM1). The study will assess walking speed, muscle strength, muscle size, myotonia, heart rhythm, mental…
- Trial name
- MOS-DM1/POP (NCT02308657)
- Where
- 6 sites in United States
- Volunteers
- 120 people
- Sponsor
- University of Rochester
Duchenne Muscular Dystrophy
Stacking Exercises Aid the Decline in FVC and Sick Time
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths…
- Trial name
- STEADFAST (NCT01999075)
- Testing
- Lung Volume Recruitment (LVR), Conventional Treatment
- Where
- 9 sites in Canada
- Volunteers
- 70 people
- Sponsor
- Children's Hospital of Eastern Ontario
Aging
A Model for Healthy Aging: Moscow Centenarians (AGE-100)
This study evaluates the associations between biomarkers of ageing, comprehensive geriatric status, telomere length and progerin level, the composition of the gut microbiota, 16sRNA metagenome sequencing in Moscow centenarians.
- Trial name
- AGE-100 (NCT02876809)
- Where
- Russia
- Volunteers
- 100 people
- Sponsor
- Pirogov Russian National Research Medical University
Muscular Dystrophy
Effect of Aquatic Therapy on Pulmonary Functions in Patients With Muscular Dystrophy
This study aimed to assess the efficacy of aquatic therapy on pulmonary functions in patients with muscular dystrophy.
- Trial name
- NCT04574934
- Testing
- aquatic therapy
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- South Valley University
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
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