Clinical trials: Muscular Atrophy
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Spinal Muscular Atrophy
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene.
- Trial name
- NCT05994950
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
Duchenne Muscular Dystrophy, Duchenne Disease
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD
- Trial name
- NCT07766980
- Testing
- VM100 Nutritional Supplement
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
Tamoxifen in Duchenne Muscular Dystrophy
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of…
- Trial name
- TAMDMD (NCT03354039)
- Testing
- Tamoxifen
- Where
- 11 sites in France, Germany and 4 more
- Volunteers
- 93 people
- Sponsor
- University Hospital, Basel, Switzerland
Duchenne Muscular Dystrophy
Safety Study of Flavocoxid in Duchenne Muscular Dystrophy
Objective of this study is to evaluate safety and tolerability of flavocoxid administered at the daily oral dose of 500 or 1000 mg/die for one year in DMD patients, alone or in association with steroids (deflazacort on alternate…
- Trial name
- NCT01335295
- Testing
- Flavocoxid
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- University of Messina
Duchenne Muscular Dystrophy
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531…
- Trial name
- NCT07209332
- Testing
- WVE-N531
- Where
- 3 sites in Jordan, United Kingdom
- Volunteers
- 175 people
- Sponsor
- Wave Life Sciences USA, Inc.
Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
Duchenne Muscular Dystrophy Heart Study
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020.
- Trial name
- DMD-HS (NCT03443115)
- Where
- France
- Volunteers
- 700 people
- Sponsor
- Hôpital Cochin
Duchenne Muscular Dystrophy, Performance
Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy
Investigator investigated that regression of hamstring flexibility and performance in children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03589612
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Hacettepe University
Cerebral Palsy, Spina Bifida
A Peer E-mentoring Intervention to Improve Employment
Despite the strong business case of hiring people with disabilities, a significant proportion of youth with disabilities leave high school and neither work nor continue their education and are unprepared to meet the demands of a…
- Conditions
- Cerebral Palsy, Spina Bifida, Juvenile Arthritis, Spinal Cord Injury
- Trial name
- NCT02522507
- Testing
- Empowering youth towards employment
- Where
- Canada
- Volunteers
- 44 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Duchenne Muscular Dystrophy (DMD), Virtual Reality
The Effect of Virtual Reality Applications on Upper Extremity Functions in Patients With Duchenne Muscular Dystrophy
This study aims to evaluate the effects of fully immersive virtual reality (VR) applications on upper extremity (UE) functions in individuals diagnosed with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT07039799
- Testing
- Virtual Reality + Conventional Physiotherapy, Conventional Physiotherapy, Formative Usability Evaluation of the VR Rehabilitation Platform
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people
- Sponsor
- Istanbul University
Dystrophinopathy, Muscular Dystrophies
VirtualPark_Pediatric
The goal of this clinical trial is to assess the effects of a dual-task, multimodal training program on slowing functional motor decline in children and adolescents with neuromuscular disorders.
- Conditions
- Dystrophinopathy, Muscular Dystrophies, Congenital Myopathies
- Trial name
- NCT07580365
- Testing
- Dual-task cycling, Standard therapy
- Where
- 4 sites in Italy
- Volunteers
- 22 people
- Sponsor
- Istituto di Sistemi e Tecnologie Industriali Intelligenti per il Manifatturiero Avanzato
Duchenne Muscular Dystrophy
Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
The proposed phase I clinical trial is a pilot study to evaluate safety and biological activity of the rAAVrh74.MCK.micro-Dystrophin vector administered by an intramuscular route.
- Trial name
- NCT02376816
- Testing
- rAAVrh74.MCK.micro-Dystrophin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Jerry R. Mendell
Duchenne Muscular Dystrophy
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT01037309
- Testing
- PRO044 SC, PRO044 IV
- Where
- 4 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne's Muscular Dystrophy (DMD)
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.
- Trial name
- NCT01995032
- Testing
- 750 mg metformin and 7.5 g L-citrulline daily p.o.
- Where
- Switzerland
- Volunteers
- 47 people
- Sponsor
- University Hospital, Basel, Switzerland
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Glycogen Storage Disease, GSD Type 0A
Rare Glycogen Storage Diseases Natural History Study
The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome and Danon…
- Conditions
- Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII
- Trial name
- NCT06795152
- Testing
- No intervention
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Duke University
Sarcopenia
Respiratory Sarcopenia in Older Adults
The objectives of this study are to describe the prevalence of respiratory sarcopenia, evaluate its pattern of evolution over a one-year follow-up, and determine the response to a therapeutic exercise program in older adults in a…
- Trial name
- NCT06548984
- Testing
- diaphragmatic exercises, Respiratory muscle training
- Where
- Spain
- Volunteers
- 50 people
- Sponsor
- Felipe León Morillas
Muscular Diseases, Spinal Muscular Atrophy
New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy
This study aims to refine the capability of MSOT to characterise muscle tissue and to determine non-invasive, quantitative biomarkers for the disease assessment in patients with spinal muscular atrophy (SMA) using Multispectral…
- Trial name
- MSOT_SMA (NCT04115475)
- Testing
- Multispectral Optoacoustic Tomography (MSOT)
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Incomplete Spinal Cord Injury, Muscular Dystrophy
Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders
Background: Cerebral palsy (CP) is the most common motor disorder in children. CP often causes crouch gait, an abnormal way of walking. Knee crouch has many causes, so no single device or approach works best for everybody.
- Conditions
- Incomplete Spinal Cord Injury, Muscular Dystrophy, Spina Bifida, Cerebral Palsy
- Trial name
- NCT01961557
- Testing
- EA-KAFO
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Institutes of Health Clinical Center (CC)