Clinical trials: Progressive Muscular Atrophy
Muscular Atrophy, Spinal
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with…
- Trial name
- STELLAR-2 (NCT07444450)
- Testing
- Salanersen
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Biogen
Spinal Muscular Atrophy (SMA)
VRehab-SMA Phase 1.2
Spinal muscular atrophy is a genetic disorder characterized by progressive muscle weakness, severely impacting patients' motor abilities.
- Trial name
- NCT07578454
- Testing
- VRehab SMA device
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University of Oxford
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
Spinal Muscular Atrophy
Spinal Muscular Atrophy and Cardiac Autonomic Function
Respiratory dysfunction is the major cause of morbidity and mortality in patients with spinal muscular atrophy (SMA). Air stacking is a clearance airway technique frequently used but its effects on cardiac autonomic function in…
- Trial name
- NCT01522079
- Testing
- Air stacking manuever
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Centro Universitário Augusto Motta
Type 2 Spinal Muscular Atrophy, Type 3 Spinal Muscular Atrophy
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
NatHis-SMA is a prospective, longitudinal and interventional study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy (SMA).
- Trial name
- NatHis-SMA (NCT02391831)
- Testing
- Strength, function and activity measurements, Muscle MRI, Electrophysiology measurements
- Where
- 9 sites in Belgium, France and 1 more
- Volunteers
- 81 people
- Sponsor
- Institut de Myologie, France
Muscular Atrophy, Spinal
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA.
- Trial name
- SUNFISH (NCT02908685)
- Testing
- Risdiplam
- Where
- 45 sites in United States, Belgium and 14 more
- Volunteers
- 231 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
Spinal Muscular Atrophy and School Transitions
A qualitative questionnaire-based study to explore the experiences of children and young people with Spinal Muscular Atrophy (SMA) and their families regarding school transition and educational participation.
- Trial name
- NCT07720518
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
Spinal Muscular Atrophy
SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients
The first cure for Spinal Muscular Atrophy (SMA; Nusinersen) has been approved by FDA in 2017. Although it improves the clinical picture of most SMA patients, not all exhibit the same response to treatment.
- Trial name
- NCT05761262
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots.
Spinal muscular atrophy (SMA) is a motor neuron disorder caused by the absence of a functional survival of motor neuron 1, telomeric (SMN1) gene. Type I SMA, a lethal disease of infancy, accounts for the majority of cases.
- Trial name
- NCT05237544
- Where
- United Kingdom
- Volunteers
- 5,065 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Spinal Muscular Atrophy
SMN Circular RNAs as Potential New Targets and Biomarkers for SMA
Spinal Muscular Atrophy (SMA) is a life-threatening disease in infancy that is caused by inactivating mutations in the Survival Motor Neuron 1 (SMN1) gene1,2.
- Trial name
- CircSMA (NCT05760209)
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
This is an open label phase I/II clinical trial to assess safety, tolerability and potential effect on SMN mRNA and protein in vivo of a compound in which preliminary evidence supports a potential effect on SMN levels in vitro.
- Trial name
- NCT00374075
- Testing
- Valproic Acid
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Utah
Spinal Muscular Atrophy
Mechanisms of Cell Death in Spinal Muscular Atrophy
Spinal muscular atrophy is a genetically based disease that affects motor neurons in the spinal cord and leads to muscle wasting and weakness.
- Trial name
- NCT01754441
- Where
- 2 sites in United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Nemours Children's Clinic
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy
AveXis Managed Access Program Cohort for Access to AVXS-101
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.
- Trial name
- NCT03955679
- Testing
- AVXS-101
- Where
- United States
- Sponsor
- United BioSource, LLC
Cerebral Palsy, Acquired Brain Injury
Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment
The goal of this clinical trial is to analyze the usability and safety of the robotic gait device EXPLORER in children with cerebral palsy, acquired brain injury and spinal muscular atrophy.
- Conditions
- Cerebral Palsy, Acquired Brain Injury, Spinal Muscular Atrophy
- Trial name
- NCT06167954
- Testing
- EXPLORER
- Where
- 5 sites in Spain
- Volunteers
- 50 people
- Sponsor
- MarsiBionics
Spinal Muscular Atrophy, Neuromuscular Disease
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
This study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01166022
- Testing
- Exercise
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Spinal Muscular Atrophy
Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
This prospective interventional study evaluates the immediate and sustained effects of intensive equine-assisted physiotherapy in children with spinal muscular atrophy (SMA).
- Trial name
- HIPOSMA (NCT05341453)
- Testing
- Individual physiotherapy (SMA-SOC-N), Equine-Assisted Physiotherapy Based on Neuroproprioceptive Facilitation and Inhibition, Therapeutic grooming
- Where
- Czechia
- Volunteers
- 21 people
- Sponsor
- Charles University, Czech Republic
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy
A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy
This is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational satisfaction of children and youth (8-18 years old…
- Trial name
- INFORM SMA (NCT06396325)
- Testing
- Tales from the Magic Keep™, Usual Care
- Where
- 5 sites in Canada
- Volunteers
- 22 people
- Sponsor
- Maryam Oskoui, MD, MSc, FRCPC, FAAN
Spinal Muscular Atrophy
Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium
Medico-economic study of Newborn screening of Spinal Muscular Atrophy
- Trial name
- NCT03554343
- Testing
- test for SMN1 exon 7 deletion
- Where
- Belgium
- Volunteers
- 136,339 people
- Sponsor
- Centre Hospitalier Régional de la Citadelle