Clinical trials: Atrophy
Duchenne Muscular Dystrophy
CoQ10 and Prednisone in Non-Ambulatory DMD
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD.
- Trial name
- NCT00308113
- Testing
- Prednisone, Coenzyme Q10
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy (DMD)
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration.
- Trial name
- Acti-nRoll (NCT07664124)
- Testing
- Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Muscular Dystrophy, Duchenne
A Study of Tadalafil for Duchenne Muscular Dystrophy
The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01865084
- Testing
- Tadalafil
- Where
- 62 sites in United States, Argentina and 14 more
- Volunteers
- 331 people
- Sponsor
- Eli Lilly and Company
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the…
- Trial name
- NCT00568698
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Stanford University
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
This is an investigation of the efficacy and safety of CRD007 in Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers.
- Trial name
- NCT01540604
- Testing
- CRD007
- Where
- Sweden
- Sponsor
- RSPR Pharma AB
Duchenne Muscular Dystrophy
Relationship Between PFTs and Pdi in DMD
A cross-sectional study to explore the relationship between clinically assessed pulmonary function test (PFT) measures and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy (DMD) as well as to explore the…
- Trial name
- NCT03335384
- Testing
- Esophageal Balloon, Gastric Balloon, Nasal Pressure Transducer
- Sponsor
- University of Minnesota
Duchenne Muscular Dystrophy
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT00428935
- Testing
- rAAV2.5-CMV-minidystrophin (d3990)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Nationwide Children's Hospital
Spinal Muscular Atrophy
A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy
This is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational satisfaction of children and youth (8-18 years old…
- Trial name
- INFORM SMA (NCT06396325)
- Testing
- Tales from the Magic Keep™, Usual Care
- Where
- 5 sites in Canada
- Volunteers
- 22 people
- Sponsor
- Maryam Oskoui, MD, MSc, FRCPC, FAAN
Duchenne Muscular Dystrophy
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Trial name
- NCT03373968
- Testing
- Givinostat
- Where
- 39 sites in United States, Belgium and 9 more
- Volunteers
- 206 people
- Sponsor
- Italfarmaco
Sarcopenia
Protein Timing, Lean Mass, Strength and Functional Capacity Gains in Postmenopausal Women
This study evaluated the effect of protein intake immediately after resistance exercise on lean mass, strength, and functional capacity gains in postmenopausal women.
- Trial name
- NCT03372876
- Testing
- Protein and carbohydrate supplementation
- Where
- Brazil
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Federal University of Uberlandia
Facioscapulohumeral Muscular Dystrophy
The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry
Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000.
- Trial name
- NCT04001582
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,018 people
- Sponsor
- Newcastle University
Spinal Muscular Atrophy
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in…
- Trial name
- SHINE (NCT02594124)
- Testing
- nusinersen
- Where
- 47 sites in United States, Australia and 12 more
- Volunteers
- 292 people
- Sponsor
- Biogen
Alveolar Bone Loss
Comparison of Coronally Advanced Lingual Flap to Modified MPI for Flap Advancement in Partially Edentulous Patients
Comparison of cronally advanced lingual flap to modified periosteal releasing incision (MPRI)
- Trial name
- NCT03466840
- Testing
- The coronally advanced lingual flap, Modified periosteal releasing Incision
- Where
- Egypt
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Cairo University
Genitourinary Syndrome of Menopause
Management of Cancer Therapy Related Vulvovaginal Atrophy
The goal of this clinical trial is to: 1. evaluate the efficacy of PRP injection and PRP injection mixed with non-cross-linked hyaluronic acid compared to the control group receiving standard therapy with topical non-cross-linked…
- Trial name
- NCT05782920
- Testing
- Platelet rich plasma, non cross-linked hyaluronic acid, topical HA gel
- Where
- Egypt
- Volunteers
- 45 people
- Sponsor
- Alexandria University
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Facioscapulohumeral Muscular Dystrophy
Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
The purpose of this research study is to identify and study changes in muscle in people with facioscapulohumeral muscular dystrophy using magnetic resonance imaging and spectroscopy.
- Trial name
- NCT01671865
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Duchenne Muscular Dystrophy (DMD)
Usability and User Experience of a Virtual Reality Rehabilitation Game Platform in Individuals With Duchenne Muscular Dystrophy
This study aims to evaluate the usability, user experience, and tolerability of a virtual reality-based rehabilitation game platform specifically developed for individuals with Duchenne muscular dystrophy.
- Trial name
- DMD-VR-UE (NCT07565272)
- Testing
- Virtual Reality Gameplay Session
- Where
- Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Istanbul University
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
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