Clinical trials: Duchenne Disease
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
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- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
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
Duchenne Muscular Dystrophy
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD…
- Trial name
- NCT02760277
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
Dilated Cardiomyopathy, Lef Ventricular Dysfunction
Prospective Becker-Heart-Study
The purpose of this study is to determine whether electrocardiogram, echocardiography, cardiac MRI, sera biomarkers can improve early detection of myocardial involvement and clinical outcome.
- Conditions
- Dilated Cardiomyopathy, Lef Ventricular Dysfunction, Heart Failure
- Trial name
- Becker-HS (NCT02020954)
- Testing
- ECG, echocardiography, cardiac MRI, sera biomarkers
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Karim WAHBI
Duchenne Muscular Dystrophy
Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study
Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding of DMD natural history. This study aim to assess the natural history of DMD through a composite assessment tool capable of capturing…
- Trial name
- NCT02780492
- Testing
- Set of assessment tools
- Where
- 5 sites in France, Netherlands and 1 more
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- University College, London
Duchenne Muscular Dystrophy, Cardiomyopathy
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age.
- Trial name
- HOPE (NCT02485938)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Capricor Inc.
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Duchenne Muscular Dystrophy
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
- Trial name
- MISSION (NCT02606136)
- Testing
- Pamrevlumab
- Where
- 10 sites in United States
- Volunteers
- 21 people
- Sponsor
- Kyntra Bio
Duchenne Muscular Dystrophy, Natural History
phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function
- Conditions
- Duchenne Muscular Dystrophy, Natural History, Motor Function; Retardation
- Trial name
- GUP21003 (NCT06366815)
- Testing
- Different Phenotypes of non ambulant patients with Duchenne Muscular Dystrophy
- Where
- 2 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Osteoporosis, Duchenne Muscular Dystrophy
Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a X-linked recessive disorder due to a mutation of the dystrophin gene (Xp21). Dystrophin is a sarcolemmal protein of skeletal and cardiac muscle, and its absence causes progressive muscle…
- Trial name
- NCT05281120
- Testing
- Low-level mechanical vibrations WITH vertical sinusoidal acceleration, Low-level mechanical vibrations WITHOUT vertical sinusoidal acceleration
- Volunteers
- 20 people
- Sponsor
- Istituto Auxologico Italiano