Clinical trials: Spinal Muscle Atrophy, open to volunteers
Spinal Muscular Atrophy (SMA)
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .
- Trial name
- NCT06888661
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 4 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS
Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity
Background: Androgen effects in humans are usually (but not always) mediated by the androgen receptor which is coded for by the androgen receptor gene (AR gene).
- Conditions
- Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
- Trial name
- NCT04708431
- Where
- United States
- Volunteers
- 650 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Spinal Muscular Atrophy
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages…
- Trial name
- SOLAR (NCT07444476)
- Testing
- Salanersen
- Where
- 3 sites in United States, China
- Volunteers
- 90 people
- Sponsor
- Biogen
Triple Negative Breast Neoplasms
Bicalutamide and Abemaciclib in Inoperable or Metastatic Androgen Receptor-positive Triple-negative Breast Cancer
This study has as goal to evaluate the use of abemaciclib and bicalutamide in androgen receptor positive metastatic triple negative breast cancer.
- Trial name
- ABBICAR (NCT06365788)
- Testing
- Abemaciclib, Bicalutamide
- Where
- 6 sites in Belgium
- Volunteers
- 53 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
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
Metastatic Castration Resistant Prostate Cancer
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
Himalayas-1 is a randomized, open-label, global, multicenter phase 3 study evaluating whether the combination of rinzimetostat with darolutamide is more effective compared to physician's choice of control; ARPI (darolutamide or…
- Trial name
- Himalayas-1 (NCT07723248)
- Testing
- Rinzimetostat, Darolutamide, Enzalutamide
- Where
- 15 sites in United States, Puerto Rico
- Volunteers
- 600 people
- Sponsor
- ORIC Pharmaceuticals
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy (SMA)
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy.
- Trial name
- BLUEBELL (NCT05747261)
- Testing
- ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
- Where
- 7 sites in Belarus, Russia
- Volunteers
- 40 people
- Sponsor
- Biocad
SMA - Spinal Muscular Atrophy
An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
This study is an open label individual patient study of the drug Nusinsersen.
- Trial name
- NCT07838779
- Testing
- Nusinersen Injectable Product
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Neuromuscular Disorders, Neurodevelopmental Disorders
Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.
Neurodevelopmental disorders often result in abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as the inability to stand and walk.
- Trial name
- NCT07159360
- Testing
- CLINICAL EXPLORER
- Where
- 2 sites in Mexico, Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- MarsiBionics
Muscular Atrophy, Spinal
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with…
- Trial name
- STELLAR-2 (NCT07444450)
- Testing
- Salanersen
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
The purpose of this project is to investigate the utility of the SMA EFFORT, an SMA-specific patient-reported outcome measure, to assess perceived physical fatigability that is anchored to intensity and duration of activities.
- Trial name
- NCT06955897
- Testing
- Observational
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Columbia University
Spinal Muscle Atrophy
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with…
- Trial name
- NCT07531719
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy (SMA)
Long Read Analysis in Spinal Muscular Atrophy - LOREASI
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disease caused by deletion of the SMN1 gene, with the most severe form leading to death in children without treatment.
- Trial name
- LOREASI (NCT07332702)
- Testing
- blood sample
- Where
- France
- Volunteers
- 27 people
- Sponsor
- University Hospital, Rouen
Anarthria, Dysarthria
Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)
The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that will enable people who have lost the ability to…
- Conditions
- Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries
- Trial name
- BG-Speech-02 (NCT06094205)
- Testing
- BrainGate Neural Interface System
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Leigh R. Hochberg, MD, PhD.
Spinal Amyotrophy, Infantile Spinal Muscular Atrophy
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by the degeneration of motor neurons in the anterior horn of the spinal cord, due to the absence of the SMN1 gene and the resulting lack of SMN…
- Conditions
- Spinal Amyotrophy, Infantile Spinal Muscular Atrophy, Juvenile Spinal Muscular Atrophy
- Trial name
- FANTASI-SMART (NCT06562283)
- Testing
- Thumb test, Grip test, Quadriceps Intermittent Fatigue test (QIF test))
- Where
- 4 sites in France
- Volunteers
- 80 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Spinal Cord Injury, Amyotrophic Lateral Sclerosis
iBCI Optimization for Veterans With Paralysis
VA research has been advancing a high-performance brain-computer interface (BCI) to improve independence for Veterans and others living with tetraplegia or the inability to speak resulting from amyotrophic lateral sclerosis…
- Conditions
- Spinal Cord Injury, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome
- Trial name
- NCT05470478
- Testing
- Mobile neural decoding platform (mobile iBCI)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- VA Office of Research and Development
Infantile Spinal Muscular Atrophy
Test of Aquatic Mobility of SMA Infants
Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death.
- Trial name
- BAINSMA (NCT06152302)
- Testing
- Mobility study in dry conditions, Mobility study in water conditions
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Secondary Osteoporosis, Sarcopenia
The Effect of Exoskeletal-assisted Walking Combined With Transcutaneous Spinal Cord Stimulation on Bone Strength.
Immobilization following spinal cord injury (SCI) results in muscle and bone loss below the level of injury, which ultimately predisposes to fracture at several sites throughout the legs and can lead to several medical…
- Trial name
- NCT07325149
- Testing
- Exoskeleton-Assisted Walking (EAW) + active Transcutaneous Spinal Cord Stimulation (tSCS)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Bronx VA Medical Center
Spinal Muscular Atrophy
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
With the advent of new treatments for ASI, new phenotypes are emerging. The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2…
- Trial name
- PHENO SMART (NCT06321965)
- Testing
- evaluation of muscle function, First-line cognitive assessment, second-line cognitive assessment
- Where
- 8 sites in France
- Volunteers
- 60 people
- Sponsor
- Hospices Civils de Lyon