Clinical trials: Spinal Muscular Atrophy
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
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
Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
Spinal Cord Stimulation in Spinal Muscular Atrophy
Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical…
- Trial name
- SCSinSMA (NCT05430113)
- Testing
- Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Marco Capogrosso
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 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
Spinal Muscular Atrophy Type I, Scoliosis
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation.
- Conditions
- Spinal Muscular Atrophy Type I, Scoliosis, Chest Deformities, Spinal Orthosis
- Trial name
- NCT05878418
- Testing
- Thoracolumbosacral Spinal Orthosis (TLSO), Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Medipol University
Infantile-onset Spinal Muscular Atrophy
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
- Trial name
- NCT02865109
- Testing
- Nusinersen
- Where
- 7 sites in Colombia, New Zealand and 1 more
- Sponsor
- Biogen
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
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT01780246
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Biogen
SMA - Spinal Muscular Atrophy
An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)
An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen
- Trial name
- NCT07846696
- Testing
- Nusinersen Injectable Product
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Spinal Muscular Atrophy
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe. We plan to work together to find the best common outcome measures for the following multicenter therapeutic…
- Trial name
- NCT01611610
- Testing
- Functional tests
- Volunteers
- 4 people
- Sponsor
- Institut de Myologie, France
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Spinal Muscular Atrophy Type III, Spinal Muscular Atrophy Type II
Spinal Cord Gray Matter Imaging in Spinal Muscular Atrophy
This study aims to measure the spinal cord gray matter in patients with spinal muscular atrophy (SMA) type II and III in comparison with age- and sex-matched healthy controls (HC) using rAMIRA (radially sampled averaged…
- Trial name
- NCT06137612
- Testing
- MRI
- Where
- Switzerland
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- University Hospital, Basel, Switzerland
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA).
- Trial name
- ENDEAR (NCT02193074)
- Testing
- nusinersen
- Where
- 31 sites in United States, Australia and 11 more
- Volunteers
- 122 people
- Sponsor
- Biogen
SMA
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a…
- Trial name
- STRIVE-EU (NCT03461289)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 10 sites in Belgium, France and 2 more
- Volunteers
- 33 people
- Sponsor
- Novartis Gene Therapies
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Study of ALS Reversals 4: LifeTime Exposures
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e.
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
- Trial name
- StARLiTE (NCT03706391)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Duke University
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders
The investigators laboratory has been studying families with a history of ALS for more than 30 years and is continuing to use new ways to understand how genes may play a role in ALS, motor neuron disease and other neuromuscular…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease
- Trial name
- NCT01459302
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- University of Massachusetts, Worcester
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