Clinical trials: Atrophy
Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy
The goal of this clinical trial is to learn if providing a multicomponent intervention improves the confidence of caregivers of children with DMD or SMA.
- Trial name
- SEE-NMD (NCT07356063)
- Testing
- Multicomponent Intervention
- Where
- Pakistan
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
Duchenne Muscular Dystrophy
Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle
The purpose of this research study is to determine the potential of Optical Imaging techniques to detect muscle damage in boys with Duchenne Muscular Dystrophy and unaffected exercised muscle.
- Trial name
- NCT02168114
- Testing
- Exercising, Optical and Magnetic Resonance Imaging and Spectroscopy
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University of Florida
Muscle Atrophy
Milk Versus a Pea-based Beverage for Bone and Muscle Health in Young Athletes
Canada's Food Guide places an increased emphasis on plant-based proteins; however, Canadians who consume a plant-based diet may be compromised because of intake of lower-quality protein.
- Trial name
- NCT05382026
- Testing
- Nutritional supplementation during a 6 month resistance training program
- Where
- Canada
- Volunteers
- 112 people, healthy volunteers welcome
- Sponsor
- University of Saskatchewan
Congenital Myotonic Dystrophy, CDM
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.
- Trial name
- ASPIRE-DM1 (NCT05224778)
- Where
- 5 sites in United States, Italy
- Volunteers
- 50 people
- Sponsor
- Virginia Commonwealth University
Infantile-onset Spinal Muscular Atrophy
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
- Trial name
- NCT02865109
- Testing
- Nusinersen
- Where
- 7 sites in Colombia, New Zealand and 1 more
- Sponsor
- Biogen
Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy
Gyrate Atrophy Ocular and Systemic Study
The Gyrate Atrophy Ocular and Systemic Study characterizes the natural history of ornithine levels and retinal degeneration (RD) associated with disease-causing OAT variants in the presence of standard care dietary treatment…
- Conditions
- Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy, Ornithine-δ-aminotransferase, OAT
- Trial name
- GYROS (NCT05312736)
- Where
- 11 sites in United States, Brazil and 6 more
- Volunteers
- 46 people
- Sponsor
- Jaeb Center for Health Research
Post-acne Atrophic Scars
Various Procedural Treatment Options for Post-acne Atrophic Scars
Post acne scars are formed as a result of very inflamed and nodulocystic acne. Most common type of acne scars are atrophic scars and they are further classified on the basis of depth and size of destruction into ice pick, boxcar…
- Trial name
- NCT06770608
- Testing
- micro-needling with topical human actrapid insulin, Micro-needling with topical Platelet rich plasma
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- Sheikh Zayed Medical College
Spinal Muscular Atrophy
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies.
- Trial name
- NCT05768048
- Where
- 5 sites in Italy
- Volunteers
- 500 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Parkinson Disease, MSA - Multiple System Atrophy
Exploring the Olfactory Mucosa, Blood and Urine for the Identification of Early Biomarkers of Parkinson's Disease, Atypical Parkinsonisms and Neurocognitive Disorders Due to Lewy Body Disease
Clinical diagnosis of Parkinson's disease (PD), multiple system atrophy (MSA) and dementia with Lewy bodies (DLB) is challenging, especially in the early stages.
- Conditions
- Parkinson Disease, MSA - Multiple System Atrophy, Lewy Body Dementia (LBD), Neurodegenerative Disease
- Trial name
- EXTRAORDINARY (NCT06846658)
- Where
- 3 sites in Italy
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Duchenne Muscular Dystrophy
Remote Physiotherapy to Protect Physical Health in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive genetic neuromuscular disease characterized by progressive loss of motor function, respiratory failure, and cardiomyopathy required regular physiotherapy.
- Trial name
- NCT06103006
- Testing
- Telerehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people
- Sponsor
- Lokman Hekim University
Duchenne Muscular Dystrophy (DMD)
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully…
- Trial name
- NCT01540409
- Testing
- AVI-4658 (Eteplirsen)
- Where
- 11 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Muscular Dystrophy, Duchenne
KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033813
- Testing
- Oxatomide (tinset)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
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
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles.
- Trial name
- GRASP-01-001 (NCT03981289)
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Virginia Commonwealth University
Duchenne Muscular Dystrophy
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
The purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.
- Trial name
- NCT00451074
- Testing
- Gentamicin infusions twice a week for six months
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Nationwide Children's Hospital
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Spinal Muscular Atrophy
Antisense Oligonucleotide for Spinal Muscular Atrophy
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.
- Trial name
- NCT05187260
- Testing
- Nusinersen
- Where
- 3 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Duchenne Muscular Dystrophy (DMD)
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02420379
- Testing
- eteplirsen
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Sarepta Therapeutics, Inc.
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