Clinical trials: BMD
Duchenne Muscular Dystrophy
Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy
This study investigates the correlation between assessments measuring functional capacity and functional capability in patients with Duchenne muscular dystrophy.
- Trial name
- NCT05249361
- Testing
- no intervention
- Where
- 2 sites in South Korea
- Volunteers
- 48 people
- Sponsor
- Samsung Medical Center
Duchenne Muscular Dystrophy
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo…
- Trial name
- NCT04740554
- Testing
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Registry for Duchenne and Becker Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- FAIR-DMD (NCT07402122)
- Where
- 30 sites in Austria, Germany
- Volunteers
- 1,500 people
- Sponsor
- Dr. Andreas Ziegler
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
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon
Osteopenia, Osteoporosis
Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation
This is a Phase 2, open-label, randomized, controlled clinical study of pediatric subjects treated with pamidronate with calcium and vitamin D versus calcium and vitamin D alone following hematopoietic cell transplantation (HCT).
- Trial name
- NCT02074631
- Testing
- Pamidronate, Calcium and vitamin D
- Where
- 2 sites in United States
- Volunteers
- 63 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Inflammatory Myopathy, Duchenne Muscular Dystrophy
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
The aim of our study is to Assess skeletal muscle structural status in children with inflammatory myositis and Duchenne muscular dystrophy using musculoskeletal ultrasound and to perform a longitudinal follow up of these changes…
- Trial name
- NCT03786913
- Testing
- Quantitative muscle ultrasound measurements
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Benha University
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.
Subclinical hypothyroïdism
Assessment of Bone Mineral Density in Patients With Subclinical Hypothyroidism
This study aims to recognize the effects of subclinical hypothyroidism on bone mineral density, Not many studies were done on this subject
- Trial name
- ABMDPSH (NCT04270110)
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Assiut University
Bone Cancer
Retrospective Study of the Biomechanical Properties of Large Allografts
Primary Objectives: 1. To evaluate the material properties, histomorphometric indices, bone mineral density (BMD), and presence of microfractures in retrieved large allograft cortical bone specimens removed from orthopaedic…
- Trial name
- NCT00632294
- Testing
- Tissue Sample
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- M.D. Anderson Cancer Center
Duchenne Muscular Dystrophy
Digestive Events in Duchenne Muscular Dystrophy Patients
Relation between clinical and genetic features and acute digestive events in Duchenne muscular dystrophy patients
- Trial name
- DIGD (NCT04384354)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Duchenne Muscular Dystrophy
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
- Trial name
- NCT04687020
- Testing
- Viltolarsen
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- NS Pharma, Inc.
Osteoarthritis of Hip
Bone Mineral Changes Around Stemless and Stemmed Cementless Femoral Components
The purpose of this study is to determine if there is a difference in stress shielding and bone resorption of the proximal femur in two anatomical stem total hip designs.
- Trial name
- NCT00957970
- Testing
- Stemless femoral component, IPS, Depuy cementless femoral stem
- Where
- South Korea
- Volunteers
- 120 people
- Sponsor
- Ewha Womans University
Spinal Cord Injury
The Effect of FES on Children With Spinal Cord Injury
Regular exercise is strongly recommended to help maintain a healthy lifestyle. Unfortunately, children and young adults with damaged spinal cords may not be able to exercise regularly.
- Trial name
- NCT00291317
- Testing
- RT 300-P FES Cycle
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Children's Specialized Hospital
Type 1 Diabetes Mellitus
Exercise Training Intervention in Children With Type 1 Diabetes
Type 1 diabetes mellitus (T1DM) is associated with multiple co-morbidities, such as hypertension, dyslipidemia, coronary heart disease and osteoporosis. The foundation of these conditions lays in childhood.
- Trial name
- Diabex (NCT01220479)
- Testing
- Exercise training program
- Where
- Switzerland
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- University Hospital, Geneva
Idiopathic Hypoparathyroidism, Autosomal Dominant Hypocalcaemia
Characterization of Patients With Non-surgical Hypoparathyroidism and Pseudohypoparathyroidism
Hypoparathyroidism (hypoPT) and pseudohypoparathyroidism (Ps-hypoPT) are rare diseases, characterized by low levels of parathyroid hormone [PTH] and plasma calcium or high plasma PTH and low plasma calcium, respectively.
- Conditions
- Idiopathic Hypoparathyroidism, Autosomal Dominant Hypocalcaemia, Pseudohypoparathyroidism
- Trial name
- NCT02551120
- Where
- Denmark
- Volunteers
- 100 people
- Sponsor
- University of Aarhus
Osteoporosis
Osteoporosis Prevention: Changes to Exercise and Diet in Children
The purpose of this study is to determine whether educating parents about health and behavior management techniques will increase physical activity, calcium intake, fitness, and bone density in their children.
- Trial name
- NCT00063050
- Testing
- Physical activity and nutrition intervention
- Where
- United States
- Volunteers
- 155 people, healthy volunteers welcome
- Sponsor
- San Diego State University
Bone Density, Gut Microbiomes
Linking Dietary Nutrition, Dietary Patterns, Gut Microbiota, and Bone Density: A Cross-Sectional Study
The study aims to understand the current situation of bone health in the Gannan Tibetan primary school student population, to explore the relationship between nutrition, intestinal microorganisms and bone health, to analyze the…
- Trial name
- NCT07256314
- Testing
- No Intervention: Observational Cohort
- Where
- China
- Volunteers
- 431 people, healthy volunteers welcome
- Sponsor
- Jianhua Ma