Clinical trials: Duchenne's Muscular Dystrophy
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.
- Trial name
- NCT06839469
- Where
- 3 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Columbia University
Duchenne Muscular Dystrophy
Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
This study, supported by Parent Project Muscular Dystrophy, will determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with Duchenne muscular dystrophy.
- Trial name
- NCT01359670
- Testing
- Tadalafil, Sildenafil
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy (DMD), Viscoelastic Property
Duchenne Muscular Dystrophy and the Viscoelastic Properties of Upper Limb Muscles
Muscular dystrophies are hereditary and progressive skeletal muscle diseases that cause degeneration and loss of strength in the muscles.
- Trial name
- NCT07435116
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Sanko University
Duchenne's Muscular Dystrophy
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD).
- Trial name
- NCT02235844
- Testing
- Umbilical Cord Mesenchymal Stem Cells
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Allergy and Asthma Consultants, Wichita, Kansas
Duchenne Muscular Dystrophy
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Researchers at Children's Hospital Boston Neurology Department invite children to participate in a new research study. Researchers are looking for boys ages 2 - 30 with Duchenne Muscular Dystrophy (DMD) and healthy boys ages 2 …
- Trial name
- QED (NCT01491555)
- Where
- United States
- Volunteers
- 73 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Osteoporosis
An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids
- Trial name
- NCT00799266
- Testing
- Zoledronic acid
- Where
- 12 sites in Australia, Canada and 4 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
DMD
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
- Trial name
- NCT07058662
- Testing
- Single dose intravenous of BBM-D101
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Belief BioMed (Beijing) Co., Ltd
Muscular Dystrophies
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876…
- Trial name
- NCT01803412
- Testing
- Drisapersen
- Where
- 13 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- BioMarin Pharmaceutical
Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy
Muscle MRI Outlining of Neuromuscular Diseases Using Artificial Intelligence
Background and aim: Neuromuscular diseases encompass a range of conditions affecting muscle cells, nerves, or the interaction between the two.
- Conditions
- Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, Hypokalemic Periodic Paralysis
- Trial name
- NCT06917430
- Testing
- No intervention
- Volunteers
- 120 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Urinary Titin Biomarker in DMD
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful…
- Trial name
- NCT07332013
- Testing
- Descending stair walk
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Duchenne Muscular Dystrophy
Upper Limb Assessment in Duchenne Muscular Dystrophy
The literature on outcome measures assessing upper limbs in Duchenne muscular dystrophy (DMD) is quite scanty. While there have been considerable advances for ambulant DMD boys, no prospective study has so far been devoted to…
- Trial name
- PUL in DMD (NCT02436720)
- Where
- Italy
- Volunteers
- 300 people
- Sponsor
- Catholic University of the Sacred Heart
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and…
- Trial name
- ESSENCE (NCT02500381)
- Testing
- SRP-4045, SRP-4053
- Where
- 76 sites in United States, Argentina and 22 more
- Volunteers
- 228 people
- Sponsor
- Sarepta Therapeutics, Inc.
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, Gamification
Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy
This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program.
- Conditions
- Duchenne Muscular Dystrophy, Gamification, Occupational Therapy
- Trial name
- DMD (NCT06402942)
- Testing
- Gamified occupational therapy programme, Classic occupational therapy programme
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Başak Çağla Arslan
Balance, Duchenne Muscular Dystrophy
The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy
Investigators investigated that the effects of kinesilogy taping on balance in patients with Duchenne Muscular Dystrophy
- Trial name
- NCT03541070
- Testing
- Kinesiology tape
- Where
- Turkey (Türkiye)
- Volunteers
- 45 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy (DMD)
DMD Voice: Qualitative Interviews With Patients and Caregivers
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.
- Trial name
- NCT06925269
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
Duchenne Muscular Dystrophy
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory…
- Trial name
- HORIZON (NCT06597656)
- Testing
- delandistrogene moxeparvovec, Plasmapheresis
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
This is an open-label, single-arm, dose-escalation trial to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular…
- Trial name
- NCT07282652
- Testing
- RAG-18
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Peking Union Medical College Hospital
Muscular Dystrophy, Duchenne Muscular Dystrophy
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
- Trial name
- BMMNC (NCT01834040)
- Testing
- Intralesional/ Intravenous of Autologous Stem cells.
- Where
- India
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Chaitanya Hospital, Pune
Becker Muscular Dystrophy
Sodium Nitrate for Muscular Dystrophy
The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation.
- Trial name
- NCT02434627
- Testing
- Sodium Nitrate
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Cedars-Sinai Medical Center