Clinical trials: Spinal Muscular Atrophy
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
Spinal Muscular Atrophy
Spinal Muscular Atrophy on Wheels, Using Power Mobility
The group of children diagnosed with Spinal Muscular Atrophy (SMA) has serious restrictions on participation. SMA is a neuromuscular disease that leads to neuromusculoskeletal disorders that limit functional activities, sometimes…
- Trial name
- SMAPmob (NCT05589987)
- Testing
- power mobility
- Where
- 2 sites in Spain
- Volunteers
- 20 people
- Sponsor
- University of Castilla-La Mancha
Spinal Muscular Atrophy
Holter of Movement in Patients with SMA Undergoing Treatment.
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam.
- Trial name
- NCT04888702
- Testing
- Actimyo
- Where
- 3 sites in Belgium, Romania
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Non Invasive Ventilation, Spinal Muscular Atrophy
Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
SMA (Spinal Muscular Atrophy) is a rare neuromuscular disease characterized by motoneuron damage. Symptoms consist of respiratory involvement with numerous respiratory infections and eventually respiratory failure, for which NIV…
- Conditions
- Non Invasive Ventilation, Spinal Muscular Atrophy, Respiratory Bacterial Infection
- Trial name
- AVNIR (NCT05712330)
- Testing
- non invasive ventilation samples, patient samples, parent questionnaire
- Where
- France
- Volunteers
- 8 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Incomplete Spinal Cord Injury, Muscular Dystrophy
Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders
Background: Cerebral palsy (CP) is the most common motor disorder in children. CP often causes crouch gait, an abnormal way of walking. Knee crouch has many causes, so no single device or approach works best for everybody.
- Conditions
- Incomplete Spinal Cord Injury, Muscular Dystrophy, Spina Bifida, Cerebral Palsy
- Trial name
- NCT01961557
- Testing
- EA-KAFO
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
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
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
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