Clinical trials: Spinal Muscle Atrophy
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)
Muscular Atrophy, Spinal
Pediatric Spinal Muscular Atrophy (SMA) China Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q…
- Trial name
- NCT05042921
- Where
- 25 sites in China
- Volunteers
- 600 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
The aim of the study is to propose a method for quantifying motor function in infants with spinal muscular atrophy treated with innovative therapies using inertial sensors.
- Trial name
- IMUSMA (NCT04833348)
- Testing
- Motor function measurement using inertial sensors
- Where
- France
- Volunteers
- 35 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia
Phenotype, Genotype and Biomarkers 2
The purpose of this study is to learn more about amyotrophic lateral sclerosis (ALS) and other related neurodegenerative diseases, including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic…
- Conditions
- Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Trial name
- PGB2 (NCT04875416)
- Where
- 4 sites in United States, South Africa
- Volunteers
- 217 people, healthy volunteers welcome
- Sponsor
- University of Miami
Cerebral Palsy, Caregiver Burden
Awareness Levels of Caregivers of Disabled Children
Disability-or apology; It is the inability or incompleteness of individuals to fulfill their roles in life, such as age, gender, culture, social and psychological factors, due to their inadequacies.
- Conditions
- Cerebral Palsy, Caregiver Burden, Disabilities Multiple, Muscular Dystrophy
- Trial name
- caregivers (NCT05228405)
- Testing
- All of the disabled children's caregivers will answer the Questionnaire of Disease, Physiotherapy and Sports Awareness
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people
- Sponsor
- Kahramanmaras Sutcu Imam University
Spinal Muscular Atrophy
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants.
- Trial name
- STOPSMA (NCT00528268)
- Testing
- Sodium phenylbutyrate
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Utah
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
This proposal will focus on (1) estimating oxidative capacity of specific muscle groups during exercise using near infrared spectroscopy and (2) describing body composition to better understand exercise capacity and mitochondrial…
- Trial name
- NCT02895789
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Columbia University
Muscular Atrophy, Spinal, Child, Only
Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy
Background and Aim: Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive symmetrical weakness and atrophy of proximal muscles causing from degeneration of anterior horn cells of spinal cord.
- Conditions
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- Trial name
- NCT05291962
- Testing
- Retrospective observational study
- Where
- Turkey (Türkiye)
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
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