Clinical trials: BMD
Duchenne Muscular Dystrophy, Exon 44
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44, DMD
- Trial name
- NCT05996003
- Testing
- NS-089/NCNP-02
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Bone Development
Bone Age of Chinese Child (BACC)
The bone age of the hand and wrist will be assessed with X-rays in more than 20,000 healthy children age 3-18 years old recruited in 28 sites from 14 province.The bone mineral density (BMD) will be measured as well.
- Trial name
- BACC (NCT04197557)
- Where
- China
- Volunteers
- 20,166 people, healthy volunteers welcome
- Sponsor
- Beijing Jishuitan Hospital
Heart Transplantation, Liver Transplantation
Zoledronic Acid Versus Alendronate for Prevention of Bone Loss After Organ Transplantation
The purpose of this study is to compare the effectiveness and safety of zoledronic acid with alendronate in the prevention of bone loss after organ transplantation. Zoledronic acid is given as a single intravenous infusion.
- Conditions
- Heart Transplantation, Liver Transplantation, Bone Resorption
- Trial name
- CTX (NCT00297830)
- Testing
- Zoledronic acid, Alendronate
- Where
- United States
- Volunteers
- 111 people
- Sponsor
- Columbia University
Osteoporosis, Nutrition
Validation of a Revised Food Frequency Questionnaire
The purpose of this study is to validate a revised food frequency questionnaire that is designed to capture vitamin D, calcium and protein intake in a population of elderly men and women with fragility fractures.
- Trial name
- FFQ (NCT02693184)
- Testing
- Full length Block 2014 Food Frequency Questionnaire, Revised Calcium, Vitamin D and Protein Screener
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- University of Nebraska
Spinal Cord Injury
Effects of Electrical Stimulation and Vitamin D Supplementation on Bone Health Following Spinal Cord Injury.
Neurogenic osteoporosis is a common complication of spinal cord injury (SCI) that is associated with low impact bone fractures. It is concerning that more than 46,000 Veterans affected with SCI and are at risk of osteoporosis and…
- Trial name
- NCT05008484
- Testing
- NMES, Vitamin D, Passive movement
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- VA Office of Research and Development
Duchenne Muscular Dystrophy (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Duchenne Muscular Dystrophy
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or…
- Trial name
- NCT04179409
- Testing
- Amondys 45, Exondys 51, Vyondys 53
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Kevin Flanigan
Hypercalciuria; Idiopathic, Nephrolithiasis
Comparison of the Impact of Diet vs Thiazide in BMD in Children With Idiopathic Hypercalciuria
The investigator's objective is to compare and evaluate the impact of nutritional treatment vs. pharmacological treatment (hydrochlorothiazide) in bone mineral density in children with idiopathic hypercalciuria.
- Trial name
- HIC (NCT03951558)
- Testing
- Diet for calciuria prevention, Hydrochlorothiazide
- Where
- Mexico
- Volunteers
- 90 people
- Sponsor
- Hospital Infantil de Mexico Federico Gomez
Muscular Dystrophy, Duchenne, Upper Extremity
The Effects of Trunk Exercises on Upper Extremity and Respiratory Functions in DMD
The aim of this study was to investigate the effects of trunk training on trunk control, upper extremity, and pulmonary function in children with Duchenne muscular dystrophy (DMD).
- Conditions
- Muscular Dystrophy, Duchenne, Upper Extremity, Respiratory Functions
- Trial name
- NCT04669847
- Testing
- Trunk-oriented exercise program, Conventional exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 26 people
- Sponsor
- Gokce Yagmur Gunes Gencer
Duchenne Muscular Dystrophy
Trial of Cell Based Therapy for DMD
This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals…
- Trial name
- NCT06692426
- Testing
- MyoPAXon, Tacrolimus
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Duchenne Muscular Dystrophy (DMD), Chronic Pain
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
1. Brief Summary This prospective observational study aims to evaluate the 6-month longitudinal changes in chronic pain, pain characteristics, coping strategies, and functional performance in children and adolescents aged 7 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Chronic Pain, Neuromuscular Diseases (NMD)
- Trial name
- NCT07892196
- Testing
- Routine Clinical Follow-up and Pain Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Antalya Training and Research Hospital
Obesity in Children, Bone Strength
Bone Metabolism in 12-21 Year Olds Undergoing Glucagon Like Peptide (GLP)-1 Receptor Agonist Therapy
The goal of this clinical trial is to compare bone health markers over 24 months in participants 12 - 21 years of age with obesity who are starting the glucagon-like peptide-1 receptor agonists (GLP-1RAs) as compared to those…
- Conditions
- Obesity in Children, Bone Strength, GLP - 1, Lifestyle Modification
- Trial name
- NCT06903923
- Testing
- GLP-1 receptor agonist, Lifestyle intervention
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- University of Virginia
Duchenne Muscular Dystrophy
Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
This is a Phase 1/2a, monocentric, open label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of SQY51 in patients with Duchenne muscular dystrophy
- Trial name
- AVANCE1 (NCT05753462)
- Testing
- Phase 1, SQY51, Phase 2a, SQY51 (cohort 1), Phase 2a, SQY51 (cohort 2)
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Sqy Therapeutics
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Pediatric Radio Frequency Coils Generic
The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).
- Trial name
- NCT01633866
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Osteoporosis, Multiple Sclerosis
Zoledronic Acid in MS-patients With Osteoporosis
This study is designed to evaluate the efficacy and safety of zoledronic acid 5 mg intravenous (i.v.) relative to placebo in Multiple Sclerosis (MS) patients with osteoporosis and to support the optimal use of zoledronic acid for…
- Trial name
- EXALT (NCT01166178)
- Testing
- Zoledronic Acid, Calcium and Vitamin D combination
- Where
- 15 sites in Germany
- Volunteers
- 29 people
- Sponsor
- Novartis
Breast Neoplasms
Exemestane and Celecoxib in Postmenopausal Women at High Risk for Breast Cancer
The primary goal of this 5-year study is to determine whether exemestane alone or in combination with celecoxib decreases breast tissue density in healthy postmenopausal women at high risk for breast cancer.
- Trial name
- NCT00073073
- Testing
- Exemestane, Calcium carbonate, Vitamin D
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Georgetown University
Duchenne Muscular Dystrophy
Motor Imagery on Children With DMD on Gait and Balance Functions
The most common muscular dystrophy among pediatric neuromuscular diseases is Duchenne Muscular Dystrophy (DMD). There is no consensus on a standardized physiotherapy and rehabilitation program or exercise prescription in DMD.
- Trial name
- NCT05601986
- Testing
- Physiotherapy and Rehabilitation Program, Additional Motor Imagery Training to Physiotherapy and Rehabilitation Program
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people
- Sponsor
- Gülsena Utku
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Duchenne Muscular Dystrophy
DMD- Interactive Virtual Reality Study
This study will help determine if an Interactive Virtual Reality system can improve the physiotherapy of young patients with Duchenne muscular dystrophy (DMD).
- Trial name
- DMD-IVR (NCT06274983)
- Testing
- IVR platform trial
- Where
- United Kingdom
- Volunteers
- 14 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Bone Loss
BONE: STAR (Switching to TAF Based Anti-Retroviral Therapy) Study
The primarily objective of this study is to assess the effect of switching from Tenofovir Disoproxil Fumurate (TDF) to TAF based ART on bone mass and turnover among women on DMPA for contraception.
- Trial name
- BONE:STAR (NCT03916328)
- Testing
- B/F/TAF, TDF/3TC/EFV or DTG or NVP, DMPA
- Where
- Uganda
- Volunteers
- 330 people
- Sponsor
- MU-JHU CARE