Clinical trials: SMA
Spinal Muscular Atrophy, Muscular Dystrophy
National Registry for Egyptian Pediatric Neuromuscular Diseases
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes
- Trial name
- NCT02124616
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Ain Shams University
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study of NIDO-361 in Patients With SBMA
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- PIONEER KD (NCT06411912)
- Testing
- NIDO-361
- Where
- 5 sites in Denmark, Italy and 2 more
- Volunteers
- 54 people
- Sponsor
- Nido Biosciences, Inc.
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Study of ALS Reversals 4: LifeTime Exposures
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e.
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
- Trial name
- StARLiTE (NCT03706391)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Duke University
Spinal Muscular Atrophy (SMA)
VRehab-SMA Phase 1.2
Spinal muscular atrophy is a genetic disorder characterized by progressive muscle weakness, severely impacting patients' motor abilities.
- Trial name
- NCT07578454
- Testing
- VRehab SMA device
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University of Oxford
Spinal Muscular Atrophy (SMA)
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Evaluate the diagnostic journey, patient experience, and disparities in the treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System.
- Trial name
- SMA (NCT07223489)
- Testing
- Telephone Interview
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Medstar Health Research Institute
Recruitment
Chinese Regional Spinal Muscular Atrophy Patient Registration Study
The primary objectives of this study are to obtain clinically meaningful data on the survival, outcomes, prognosis and treatment effect of all the patients with spinal muscular atrophy (SMA) 5q types 1 to 3 (according to…
- Trial name
- NCT05631418
- Where
- China
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Spinal Muscular Atrophy
Implementation of FEES in Spinal Muscle Atrophy
The major aim of this project is to assess comprehensively frequency and extent of dysphagia and bulbar dysfunction in SMA1, 2, and 3 patients by applying FEES and validated dysphagia scores.
- Trial name
- DYS-SMA (NCT04773470)
- Testing
- Flexible Endoscopic Evaluation of Swallowing
- Where
- Germany
- Volunteers
- 79 people
- Sponsor
- University of Giessen
Spinal Muscular Atrophy (SMA)
Adult SMA Research and Clinical Hub
Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA).
- Trial name
- REACH (NCT06978985)
- Testing
- Risdiplam, Nusinersen Injectable Product
- Where
- 18 sites in United Kingdom
- Volunteers
- 600 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Spinal Muscular Atrophy
Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA
The goal of this observational study is to to establish profiles of clinical progression in patients affected by the different types of SMA (type I, II and III) treated with the currently approved drugs using a structured battery…
- Trial name
- NCT05866939
- Volunteers
- 400 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) is neurodegenerative disease of anterior horn cells of spinal cord and represents the second more frequent pathology in childhood.
- Trial name
- NCT00961103
- Where
- Italy
- Volunteers
- 80 people
- Sponsor
- Azienda USL Reggio Emilia - IRCCS
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Spinal Muscular Atrophy
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
This is an observational study to investigate the improvement of NMJ defects in adult patients with SMA following treatment with Risdiplam.
- Trial name
- NCT05219487
- Testing
- Risdiplam
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Bakri Elsheikh
Spinal Muscular Atrophy, Amyotrophic Lateral Sclerosis
Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease
OBJECTIVES: I. Evaluate the effects of androgen suppression with leuprolide and androgen replacement with testosterone enanthate on muscle strength in men with Kennedy's disease or other motor neuron disease.
- Conditions
- Spinal Muscular Atrophy, Amyotrophic Lateral Sclerosis, Spinobulbar Muscular Atrophy
- Trial name
- NCT00004771
- Testing
- leuprolide, testosterone
- Volunteers
- 40 people
- Sponsor
- National Center for Research Resources (NCRR)
Muscular Atrophy, Spinal
Pilot Study of Growth Hormon to Treat SMA Typ II and III
The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.
- Trial name
- NCT00533221
- Testing
- somatotropin
- Where
- 6 sites in Germany
- Volunteers
- 20 people
- Sponsor
- University Hospital Freiburg
Covid19, Androgen Receptor Gene Overexpression
COVID-19 In-vitro Diagnostic Test and Androgen Receptor Gene Expression
This research study will evaluate the association of Androgen Receptor (AR) gene expression and COVID-19 disease severity and mortality.
- Trial name
- NCT04530500
- Testing
- COVID-19 Androgen Sensitivity Test (CoVAST)
- Where
- 2 sites in United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- University of California, Irvine
Spinal and Bulbar Muscular Atrophy (SBMA), Motor Neuron Disease
Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy
Background: Spinal and bulbar muscular atrophy (SBMA) is an inherited disease. It causes weakness in muscles used for swallowing, breathing, and speaking. SBMA mainly affects men, but women can carry the gene for it.
- Trial name
- NCT02124057
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cerebral Palsy, SMA II
Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Diseases
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused…
- Conditions
- Cerebral Palsy, SMA II, Neuromuscular Diseases
- Trial name
- NCT04837157
- Testing
- Exoskeleton rehabilitation
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- MarsiBionics
Motor Neuron Disease
Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy
Background: -Spinal and bulbar muscular atrophy (SBMA) is an inherited disorder that affects men. People with SBMA often have weakness throughout the body, including the muscles they use for swallowing, breathing, and speaking.
- Trial name
- NCT01369901
- Testing
- A functional exercise program, A stretching exercise program
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)