Clinical trials: Muscular Atrophy, Spinal, open to volunteers
Duchenne Muscular Dystrophy, Duchenne Disease
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved…
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children
- Trial name
- NCT06290713
- Testing
- Tadalafil, Exercise Training
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Florida
Muscular Atrophy, Spinal
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA.
- Trial name
- NCT05789758
- Testing
- No Intervention
- Where
- 14 sites in United States, United Kingdom
- Volunteers
- 20 people
- Sponsor
- Biogen
Spinal Muscular Atrophy (SMA)
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
Active-NBS is a study to evaluate the muscle development of patients with spinal muscular atrophy (SMA) who are diagnosed at birth. Medicines have become available in the last decade, and many patients are treated very early.
- Trial name
- Active-NBS UK (NCT07543003)
- Where
- United Kingdom
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Muscle Weakness, Problems Moving Their Arms and Legs
Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults
People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and legs.
- Trial name
- NCT06998134
- Testing
- Extension assist knee ankle foot orthosis (EA-KAFO)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Spinal Muscular Atrophy (SMA)
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it.
- Trial name
- Sparkle (NCT07287982)
- Testing
- ARGX-119 IV
- Where
- 28 sites in United States, Australia and 2 more
- Volunteers
- 60 people
- Sponsor
- argenx
Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis
Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS
CAPTURE ALS is a long-term data and biorepository platform that will facilitate future ALS research. CAPTURE ALS will provide the standardized systems and tools necessary to collect, store, and analyze vast amounts of multimodal…
- Conditions
- Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Degeneration
- Trial name
- CAPTURE ALS (NCT05204017)
- Where
- 4 sites in Canada
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type I
Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
The systematic inclusion of spinal muscular atrophy (SMA) in France's neonatal genetic screening (NGS) program, scheduled for September 2025, represents a major milestone in public health.
- Trial name
- PSYSMA (NCT07208903)
- Testing
- focus group discussions, Interview with the psychologist
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Spinal Muscular Atrophies
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
- Trial name
- ARISER (NCT06019637)
- Testing
- Onasemnogene Abeparvovec
- Where
- 2 sites in Brazil
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cerebral Palsy, Muscular Dystrophy
Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders
Background: People with cerebral palsy, spina bifida, muscular dystrophy, or spinal cord injury often have muscle weakness and problems controlling how their legs move. This can affect how they walk.
- Conditions
- Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Incomplete Spinal Cord Injury
- Trial name
- NCT05726591
- Testing
- EA-KAFO
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy
Gait and Bone Health in SMA
The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT.
- Trial name
- NCT07400198
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Jacqueline Montes
Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA)…
- Trial name
- SMA-REAL (NCT07737756)
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
Spinal and Bulbar Muscular Atrophy
Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy
The purpose of this clinical trial is to evaluate the efficacy and safety of mexiletine hydrochloride in patients with spinal and bulbar muscular atrophy.
- Trial name
- Med-SBMA (NCT06862596)
- Testing
- Mexiletine hydrochloride
- Where
- 5 sites in Japan
- Volunteers
- 68 people
- Sponsor
- Masahisa Katsuno
Spinal and Bulbar Muscular Atrophy
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
There is no cure to arrest or delay SBMA progression. It is estimated that ~1000 individuals are affected by SBMA in Italy at any given time (prevalence: 1.5/100000) with an annual incidence of 0.19/100000 males.
- Trial name
- BetaSBMA (NCT06169046)
- Testing
- Clenbuterol
- Where
- Italy
- Volunteers
- 90 people
- Sponsor
- Gianni Soraru
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
ALS, PLS
ALS/MND Natural History Study Data Repository
This is a data repository for multi-site multi-protocol clinic-based Natural History Study of ALS and Other Motor Neuron Disorders (MND).
- Conditions
- ALS, PLS, MND (Motor Neurone DIsease), Kennedy Disease
- Trial name
- NCT05966038
- Where
- 18 sites in United States, Israel and 1 more
- Volunteers
- 5,000 people
- Sponsor
- Massachusetts General Hospital
SMA
A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients
A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization…
- Trial name
- SMArt (NCT06322654)
- Testing
- Robotically Assisted Locomotion, Robotically Assisted Verticalization
- Where
- Poland
- Volunteers
- 200 people
- Sponsor
- Wiktor Dega University Orthopedic and Rehabilitation Hospital
ALS, SCI - Spinal Cord Injury
ECoG BMI for Motor and Speech Control
Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurological disorders.
- Conditions
- ALS, SCI - Spinal Cord Injury, Stroke, Multiple Sclerosis
- Trial name
- BRAVO (NCT03698149)
- Testing
- PMT/Blackrock Combination Device
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Karunesh Ganguly
Spinal Muscular Atrophy, Spine Deformity
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
- Conditions
- Spinal Muscular Atrophy, Spine Deformity, Scoliosis
- Trial name
- PIERRE (NCT05866419)
- Testing
- ThecaFlex DRx System
- Where
- 21 sites in United States, Germany and 3 more
- Volunteers
- 90 people
- Sponsor
- Alcyone Therapeutics, Inc
Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD)
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy…
- Trial name
- Active-NBS BE (NCT07286565)
- Testing
- MAIJU, Syde, Questionnaires
- Where
- Belgium
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Liege