Clinical trials: Spinal Muscular Atrophy
Spinal Muscular Atrophy
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
- Trial name
- SYNAPSE-SMA (NCT05794139)
- Testing
- NMD670
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- NMD Pharma A/S
Spinal Muscular Atrophy 1
Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
Spinal Muscular Atrophy Type 1 (SMA )Type 1 is a severe, early-onset neuromuscular condition that typically leads to profound weakness and impaired bulbar function-affecting swallowing, feeding, speech, and airway protection.
- Trial name
- NCT07596277
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Spinal Muscular Atrophy, Neuromuscular Diseases
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec…
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
- Trial name
- RESILIENT (NCT05337553)
- Testing
- taldefgrobep alfa
- Where
- 53 sites in United States, Belgium and 7 more
- Volunteers
- 269 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
Spinal and Bulbar Muscular Atrophy
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
- Trial name
- NCT03555578
- Testing
- Leuprorelin Acetate
- Where
- Japan
- Volunteers
- 1,905 people
- Sponsor
- Takeda
Spinal Muscular Atrophy
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ASCEND (NCT05067790)
- Testing
- Nusinersen
- Where
- 22 sites in United States, Germany and 3 more
- Volunteers
- 45 people
- Sponsor
- Biogen
Spinal Muscular Atrophy (SMA)
Robot-assisted Training
The goal of this clinical trial is to evaluate the long-term effects of isokinetic rehabilitation training in patients with spinal muscular atrophy (SMA).
- Trial name
- NCT06648486
- Testing
- With the assistance of wearable isokinetic training robot （ 7 children with SMA type II）
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Peking University Third Hospital
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
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
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
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
SMA - Spinal Muscular Atrophy
Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a genetic disorder that is known to cause damage to specialised nerve cells, called motor neurons.
- Trial name
- SMA-ABC (NCT07530926)
- Where
- United Kingdom
- Volunteers
- 550 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Neuromuscular Disease, Duchenne Muscular Dystrophy
Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback
Our study is a randomized controlled study and the subjects included in the study will be divided into three groups as virtual reality training, biofeedback training, and conventional rehabilitation.
- Conditions
- Neuromuscular Disease, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Virtual Reality
- Trial name
- NCT03689660
- Testing
- Virtual Reality Training, Biofeedback Training, Conventional rehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 24 people
- Sponsor
- Merve Kurt
Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis)
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark.
- Conditions
- Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis), Spinal Cord Injuries (SCI), Duchenne Muscular Dystrophy (DMD)
- Trial name
- HOMEVENT DK (NCT07467187)
- Where
- Denmark
- Volunteers
- 450 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy…
- Conditions
- Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP), Duchenne Muscular Dystrophy (DMD)
- Trial name
- NCT07254988
- Testing
- Meal test, GIP + GLP-2
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- University of Copenhagen
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 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
Muscular Atrophy, Spinal
A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
- Trial name
- NCT07474311
- Where
- Czechia
- Volunteers
- 213 people
- Sponsor
- Novartis Pharmaceuticals
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
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