Clinical trials: Atrophy
Non Invasive Prenatal Diagnosis
NIPD on CFTC for Triplet Repeat Diseases
The purpose of this study is to develop and validate an analytical and clinical NIPD test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the…
- Trial name
- DIACCIMEX (NCT03087526)
- Testing
- Non invasive prenatal diagnosis
- Where
- 8 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Spinal Muscular Atrophy
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ASCEND (NCT05067790)
- Testing
- Nusinersen
- Where
- 22 sites in United States, Germany and 3 more
- Volunteers
- 45 people
- Sponsor
- Biogen
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
Geographic Atrophy, Macular Degeneration
A Study to Learn How Avacincaptad Pegol (Izervay™) is Used in Clinical Practice in People Who Have Geographic Atrophy
This study is for people who have geographic atrophy due to age-related macular degeneration (AMD). AMD happens when the macula, the light-sensitive layer at the back of the eye called the retina, becomes damaged and causes a…
- Trial name
- NCT06779773
- Testing
- Avacincaptad pegol (ACP)
- Where
- 58 sites in United States
- Volunteers
- 488 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Genitourinary Syndrome of Menopause, Vaginal Atrophy
Vaginal Laser Therapy in Breast Cancer Survivors
This study evaluates the efficacy of vaginal CO2 laser therapy in breast cancer survivors with symptoms of Genitourinary Syndrome of Menopause.
- Conditions
- Genitourinary Syndrome of Menopause, Vaginal Atrophy, Breast Cancer, Dyspareunia
- Trial name
- NCT03738605
- Testing
- Microablative Fractional CO2 Laser Therapy
- Where
- Greece
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- National and Kapodistrian University of Athens
Gingival Recession
Hyaluronic Acid With Connective Tissue Grafting for Gingival Recession
This study evaluates whether adding 0.8% hyaluronic acid (HA) to connective tissue grafting and coronally advanced flap surgery improves clinical and esthetic outcomes in the treatment of gingival recession.
- Trial name
- NCT07291583
- Testing
- Hyaluronic Acid (0.8%) - Applied over CTG before flap closure (Test group only)., Connective Tissue Graft + Coronally Advanced Flap (CTG + CAF)
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Saglik Bilimleri Universitesi
Spinal Muscular Atrophy
Holter of Movement in Patients with SMA Undergoing Treatment.
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam.
- Trial name
- NCT04888702
- Testing
- Actimyo
- Where
- 3 sites in Belgium, Romania
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Muscular Dystrophy
Clinical Trial Readiness for the Dystroglycanopathies
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials.
- Trial name
- NCT00313677
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Katherine Mathews
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Duchenne Muscular Dystrophy
Treatment Effect of Tamoxifen on Patients With DMD
Duchenne muscular dystrophy (DMD) is a progressive devastating disease that affects mainly boys, with an incidence of about 1:3,500 live births.
- Trial name
- NCT02835079
- Testing
- Tamoxifen
- Volunteers
- 19 people
- Sponsor
- Hadassah Medical Organization
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Duchenne Muscular Dystrophy
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01847573
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Processa Pharmaceuticals
Parkinson Disease, Multiple System Atrophy
Facilitating Diagnostics and Prognostics of Parkinsonian Syndromes Using Neuroimaging
The goals of this study are: 1) to identify biomarkers using neuroimaging that are associated with progression rate using statistical methods, and 2) to identify biomarkers that are associated with the differential diagnosis of…
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- NCT03872102
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Duchenne Muscular Dystrophy
Once Weekly Infant Corticosteroid Trial for DMD
The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
- Trial name
- NCT05412394
- Testing
- Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 26 people
- Sponsor
- Anne M. Connolly
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD.
- Trial name
- NCT03648827
- Testing
- Ataluren
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Bicycle Ergometer Training in Duchenne Muscular Dystrophy
The progressive muscle weakness and contractures of the patients adversely affect their gait and balance. It is known that the disorder of the patients' balance and gait affects their functional capacity.
- Trial name
- BETDMD (NCT05330195)
- Testing
- Bicycle Ergometer Training, Home-based exercise group
- Where
- Turkey (Türkiye)
- Volunteers
- 23 people
- Sponsor
- Hacettepe University
ACL Reconstruction, ACL Surgery
Post Operative Use of Neuromuscular Electrical Stimulation (NMES) Device for ACLR Patients
This study is being conducted to learn about how neuromuscular electrical stimulation (NMES) in addition to standard of care aids in the recovery of muscle strength in patients undergoing ACLR procedure.
- Conditions
- ACL Reconstruction, ACL Surgery, ACL Injury, Electrotherapy
- Trial name
- NCT07171346
- Testing
- Zynex NexWave Electrotherapy Device
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Kansas Medical Center
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases.
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Trial name
- Dystro-miR1 (NCT07415837)
- Testing
- dosage of blood biomarker miR1
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Sarcopenia, Acute Lymphoblastic Leukemia
Physical Activity and Exercise During Early Treatment Phases for Childhood Acute Lymphoblastic Leukaemia to Protect Against Muscle Loss and Improve Frailty Outcomes
This is a small trial testing out a new approach before doing a bigger study. Researchers are observing a group of children/adolescents (ages 5-17) with acute lymphoblastic leukemia (ALL) and testing a physical activity and…
- Trial name
- PROTECT (NCT07325305)
- Testing
- Exercise
- Where
- Australia
- Volunteers
- 60 people
- Sponsor
- Murdoch Childrens Research Institute
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