Clinical trials: Muscular Dystrophies
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.
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
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
Muscular Dystrophies
Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)
This is a study to determine whether albuterol or oxandrolone, alone or in combination, are able to increase strength and muscle mass in patients with FSHD.
- Trial name
- NCT00027391
- Testing
- Albuterol, Oxandrolone
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- FDA Office of Orphan Products Development
Facio-Scapulo-Humeral Dystrophy
Computerized Facial Recognition for Automated Diagnosis of the Facio-Scapulo-Humeral Muscular Dystrophy (FSMHD)
The clinical diagnosis of Facio-Scapulo-Humeral Muscular Dystrophy (FSHMD) requires the movement of patients to a medical centre and a lengthy examination involving medical personnel, and may be underestimated in the most…
- Trial name
- CV4DIAGNOSIS (NCT04377217)
- Testing
- Video recording
- Where
- France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Nice
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
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A
Clinical and Biochemical Features for the Identification of Dominant Calpainopathies
Mutations in the CAPN3 gene cause muscular dystrophies with dysfunction in calpain-3. Calpainopathies are usually inherited in an autosomal recessive manner but in some families they can occur in a dominant inheritance.
- Trial name
- DOM-CAL (NCT05956132)
- Testing
- retrospective study
- Where
- 2 sites in Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS San Camillo, Venezia, Italy
Muscular Dystrophies, Muscle Inflammation
Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies
The study aimed to assess the safety and, partially, the efficacy of dietary supplementation of a flavonoids-, DHA- and EPA-based natural supplement in non-ambulant DMD boys and in a cohort of LGMD and FSHD patients to compare…
- Trial name
- PRO1 (NCT03317171)
- Testing
- Flavonoids, DHA, EPA
- Where
- Italy
- Volunteers
- 29 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Myotonic Dystrophy 1
DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
For some diseases, regular respiratory muscle training could delay the start of ventilation. For DM1, however, there are no clinically high-quality studies.
- Trial name
- DM-IMT (NCT04052958)
- Testing
- IMT - inspiratory muscle training
- Where
- Germany
- Volunteers
- 45 people
- Sponsor
- LMU Klinikum
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
Muscular Dystrophies, Congenital Myopathy
Bullying in Youth With Muscular Dystrophy and Congenital Myopathies
Bullying is an epidemic in Canada, and rates may be underreported. Youth with a disability were more likely to be bullied that those without disabilities, specifically if the disability was visible.
- Trial name
- NCT04733976
- Where
- 2 sites in Canada
- Volunteers
- 29 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Limb-girdle Muscular Dystrophy
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy…
- Trial name
- NCT04475926
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 205 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Facioscapulohumeral
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
- Trial name
- MOVE Peds (NCT06847282)
- Where
- 7 sites in United States, Australia
- Volunteers
- 80 people
- Sponsor
- University of Kansas Medical Center