Clinical trials: Spinal Muscular Atrophy
Post-Poliomyelitis Syndrome
Study of Mental Fatigue in Polio Survivors
Post-Poliomyelitis Syndrome (PPS) is the term describing the new problems affecting polio survivors many years after recovery from paralytic polio. Among the symptoms, fatigue is one of the most frequent and debilitating.
- Trial name
- NCT00487487
- Where
- United States
- Sponsor
- Uniformed Services University of the Health Sciences
Lumbar Spinal Stenosis (LSS)
Association Between Lumbar Muscle Atrophy, Sagittal Pelvic Alignment and Stenosis Grade in Patients With Degenerative Lumbar Spinal Stenosis
This study is to evaluate the correlation between muscle atrophy (MA), sagittal alignment, and stenosis degree in patients with lumbar spinal Stenosis (LSS).
- Trial name
- AtrophyLSS (NCT04444739)
- Testing
- radiographic data collection
- Where
- Switzerland
- Volunteers
- 165 people
- Sponsor
- University Hospital, Basel, Switzerland
SBMA, Spinal and Bulbar Muscular Atrophy (SBMA)
International SBMA Project (KDA)
This project aims at: connecting the existing registries and databases across different countries by comparing the information collected, the administered scales, the biochemical investigations performed; performing a common…
- Trial name
- KDA (NCT07443449)
- Where
- Italy
- Volunteers
- 700 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Spinal Muscular Atrophy (SMA)
Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA
This randomized, within-participant, contralateral-limb-controlled clinical trial will compare individually optimized compliant and rigid stiffness settings of a portable isokinetic knee training robot in children with spinal…
- Trial name
- NCT07822711
- Testing
- Portable Isokinetic Knee Training Robot With an Individually Optimized Compliant Setting, Portable Isokinetic Knee Training Robot With a Rigid Setting
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Peking University Third Hospital
Motor Neuron Disease
A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy…
- Trial name
- NCT03196375
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 35 sites in United States
- Volunteers
- 54 people
- Sponsor
- Flex Pharma, Inc.
Spinal and Bulbar Muscular Atrophy, Healthy Subjects
High Intensity Training in Patients With Spinal and Bulbar Muscular Atrophy
We want investigate if high intensity training can increase daily functionality without causing muscle damage in patients Spinal and Bulbar Muscular Atrophy .
- Trial name
- HIT in Kennedy (NCT02156141)
- Testing
- Supervised high intensity training, Optional training, Control period
- Where
- Denmark
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Karen Brorup Heje Pedersen
Spinal Muscular Atrophy
Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).
The goal of this study is to investigate the acceptability, feasibility, safety and efficacy of an optimized rehabilitation program for treated patients with spinal muscular atrophy (SMA) compared to the current rehabilitation…
- Trial name
- ACE SMA (NCT06419322)
- Testing
- Optimized rehabilitation program
- Where
- United Kingdom
- Volunteers
- 14 people
- Sponsor
- University of Oxford
Spinal Muscular Atrophy 1
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
- Trial name
- NCT06191354
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Kun Sun
Circulating Tumor Cell, Metastatic Prostate Cancer
Cabazitaxel in mCRPC Patients With AR-V7 Positive Circulating Tumor Cells (CTCs)
After failure on docetaxel, which has been the standard first line therapy for patients with metastatic castration-resistant prostate cancer (mCRPC), several treatment options are currently available.
- Trial name
- CABA-V7 (NCT03050866)
- Testing
- Cabazitaxel, Antihistamine, Corticosteroid
- Where
- 9 sites in Netherlands
- Volunteers
- 140 people
- Sponsor
- Erasmus Medical Center
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
Pompe Disease (Late-onset), Inclusion Body Myositis
Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
The primary aim of this nationwide, explorative, cross-sectional study in Germany is to characterize the prevalence, severity and quality of musculoskeletal pain in adult patients with late-onset Pompe disease (LOPD).
- Conditions
- Pompe Disease (Late-onset), Inclusion Body Myositis, Spinal Muscular Atrophy Type 3, FSHD
- Trial name
- NCT05272969
- Testing
- Beck depression inventory fast screen (Questionnaire), Brief Pain Inventory (BPI) (Questionnaire), German Pain Inventory (Questionnaire)
- Where
- Germany
- Volunteers
- 95 people
- Sponsor
- LMU Klinikum
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.
- Trial name
- NCT06839469
- Where
- 3 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Columbia University
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
Psoriatic Arthritis
Evaluation of Sarcopenia and Related Factors in Patients Diagnosed With Psoriatic Arthritis
The purpose of the study is identify the prevalence of sarcopenia and its associated factors in patients with psoriatic arthritis.
- Trial name
- NCT06437392
- Where
- Turkey (Türkiye)
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
Postpolio Syndrome
Effectiveness of Transcranial Magnetic Stimulation Treatment in Patients Diagnosed With Postpolio Syndrome
This randomized, double-blind, sham-controlled trial evaluated the effects of low-frequency repetitive transcranial magnetic stimulation (rTMS) over the primary motor cortex on walking capacity, fatigue, post-polio-related…
- Trial name
- NCT06926270
- Testing
- Repetitive transcranial magnetic stimulation (rTMS), Individualized home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 28 people
- Sponsor
- Ege University
SMA - Spinal Muscular Atrophy
HABIT-ILE + FST in Children With SMA
This single-arm pilot study will assess the feasibility and preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving…
- Trial name
- NCT07223320
- Testing
- Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Teachers College, Columbia University
Infantile Spinal Muscular Atrophy
Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II
The new NAVA® ventilatory mode with the SERVO-i ventilator (Maquet®) uses the electrical activity of the diaphragm (EADi) as a marker for triggering the respiratory cycle.
- Trial name
- NAVASI (NCT03395795)
- Testing
- NAVA
- Where
- France
- Volunteers
- 1 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Respiratory Failure
Respiratory Care in Intensive Care Unit for Neuromuscular Patients
Retrospective multicentric observational study exploring the population of adult neuromuscular patients presenting a respiratory distress requiring their admission in an Intensive Care Unit (ICU) for ventilatory support.
- Trial name
- NCT02936908
- Testing
- Ventilatory support
- Where
- 4 sites in France
- Volunteers
- 300 people
- Sponsor
- Hospices Civils de Lyon
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)
ALS
Genomic Translation for Amyotrophic Lateral Sclerosis Care
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms…
- Trial name
- GTAC (NCT02795897)
- Where
- 14 sites in United States, United Kingdom
- Volunteers
- 254 people
- Sponsor
- Columbia University