Clinical trials: SMA
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
SMA II
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
- Trial name
- NCT05901987
- Testing
- GC101
- Where
- 5 sites in China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Spinal Muscular Atrophy (SMA)
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration.
- Trial name
- STIM-SMA (NCT06977269)
- Testing
- High-frequency repetitive transcranial magnetic stimulation, lumbar puncture
- Where
- Ukraine
- Volunteers
- 20 people
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
SMA - Spinal Muscular Atrophy
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen.
- Trial name
- NCT06971094
- Testing
- GC101 adeno-associated virus injection
- Where
- 7 sites in China
- Volunteers
- 50 people
- Sponsor
- GeneCradle Inc
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 (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
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
Spinal Muscular Atrophy (SMA)
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor…
- Trial name
- NCT04174157
- Testing
- Prospective observational registry, Zolgensma
- Where
- 99 sites in United States, Greece and 9 more
- Volunteers
- 700 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy (SMA), Neuromuscular Disease
Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy
This study aims to investigate the relationship between ultrasonographic muscle thickness measurement and echogenicity assessment with functional scales in children with spinal muscular atrophy (SMA).
- Trial name
- NCT06599606
- Testing
- Ultrasonographic Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Istanbul University - Cerrahpasa
SMA
A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients
A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization…
- Trial name
- SMArt (NCT06322654)
- Testing
- Robotically Assisted Locomotion, Robotically Assisted Verticalization
- Where
- Poland
- Volunteers
- 200 people
- Sponsor
- Wiktor Dega University Orthopedic and Rehabilitation Hospital
SMA II, Cerebral Palsy
Paracetamol Study in Patients With Low Muscle Mass
To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.
- Trial name
- NCT03648658
- Testing
- Paracetamol 120Mg/5mL Oral Suspension
- Where
- Denmark
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Mette Cathrine Oerngreen
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
Neuromuscular Diseases, Cerebral Palsy
Rehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral Palsy
The purpose of this multicentric study is to assess the rehabilitative effect derived from the use of the ATLAS exoskeleton in children with neuromuscular diseases or cerebral palsy and with a level of less than 3 or less in the…
- Conditions
- Neuromuscular Diseases, Cerebral Palsy, SMA II
- Trial name
- NCT04813601
- Testing
- ATLAS 2030
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- MarsiBionics
Cerebral Palsy, SMA II
Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Diseases
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused…
- Conditions
- Cerebral Palsy, SMA II, Neuromuscular Diseases
- Trial name
- NCT04837157
- Testing
- Exoskeleton rehabilitation
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- MarsiBionics
Tremor, Involuntary Movements
Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen
Hyperkinetic movement disorders in patients with diseases of motor neurons will be studied. Patients with spinal muscular atrophy (SMA) and motor neuron disease patients will be studied.
- Conditions
- Tremor, Involuntary Movements, SMA, MND (Motor Neurone DIsease)
- Trial name
- NCT04825119
- Testing
- Nusinersen
- Where
- Slovenia
- Volunteers
- 110 people
- Sponsor
- University Medical Centre Ljubljana
Spinal Muscular Atrophy (SMA)
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Several factors make the use of celecoxib in human SMA patients appealing including: 1) low-dosing required for potential therapeutic effect (the corresponding dose in humans is much lower than that commonly used in adults and…
- Trial name
- SMA (NCT02876094)
- Testing
- celecoxib
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- Hugh McMillan
Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Spinal muscular atrophy (SMA) is the leading genetic cause of death of infants. Strong preclinical evidence suggests that effective therapy must be delivered as early as possible to prevent progression of the disease.
- Trial name
- NCT01736553
- Where
- 15 sites in United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Spinal Muscular Atrophy, SMA
SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4
A trial investigating the effects of pyridostigmine (mestinon) versus a placebo in a double-blind cross over trial in patients with hereditary proximal spinal muscular atrophy (SMA) types 2, 3 and 4.
- Conditions
- Spinal Muscular Atrophy, SMA, Kugelberg-Welander Disease
- Trial name
- SPACE (NCT02941328)
- Testing
- Pyridostigmine
- Where
- Netherlands
- Volunteers
- 39 people
- Sponsor
- UMC Utrecht
Natural History of Type 1 Spinal Muscular Atrophy (SMA)
Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
Spinal muscular atrophy (SMA) is an autosomal recessive disorder characterized by degeneration of motor neurons in the spinal cord and caused by mutations of the survival motor neuron 1 (SMN1) gene.
- Trial name
- NCT02466529
- Volunteers
- 111 people
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital