Clinical trials: Spinal Muscular Atrophy
Spinal Muscular Atrophy
Personalized Medicine for SMA: a Translational Project
Major breakthroughs in the treatment for Spinal muscular atrophy (SMA) have been recently achieved with various therapeutic approaches that increase full-length SMN protein levels.
- Trial name
- NCT05779956
- Where
- 2 sites in Italy
- Volunteers
- 90 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Healthy, Neuromuscular Disease
Electromyography to Diagnose Neuromuscular Disorders
This study will investigate problems with muscle weakness and control using electromyography-a test of nerve-muscle cell communication.
- Conditions
- Healthy, Neuromuscular Disease, Postpoliomyelitis Syndrome
- Trial name
- NCT00004553
- Testing
- Electromyography
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock
Spinal Muscular Atrophy (SMA)
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA.
- Trial name
- NCT07265232
- Testing
- vesemnogene lantuparvovec
- Where
- Indonesia
- Sponsor
- Lantu Biopharma
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
Muscular Atrophy, Spinal
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of…
- Trial name
- NCT02628743
- Testing
- Olesoxime
- Where
- 24 sites in Belgium, France and 5 more
- Volunteers
- 131 people
- Sponsor
- Hoffmann-La Roche
Poliomyelitis, Postpoliomyelitis Syndrome
Study of "Post-Polio Syndrome"
Polio or poliomyelitis is the disease caused by the poliovirus. The virus attacks cells in the spinal cord and causes symptoms of fever, sore throat, headache, vomiting, and stiffness of the neck.
- Trial name
- NCT00001185
- Where
- United States
- Volunteers
- 360 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinal Muscular Atrophy, Merosin Deficient Congenital Muscular Dystrophy
Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass
In a study from 2003 the investigators showed that adult patients with very low skeletal muscle mass (spinal muscular atrophy (SMA) type II, Duchenne muscular dystrophy, congenital muscular dystrophy) are prone to develop…
- Trial name
- NCT03970135
- Testing
- Fasting
- Where
- Denmark
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Amyotrophy
SMAdult - Monitoring of Movement on Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disease causing progressive muscle weakness related to degeneration of motor neurons.
- Trial name
- SMAdult (NCT07894185)
- Testing
- wearable sensors
- Where
- Belgium
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or…
- Trial name
- STRENGTH (NCT05386680)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy Type 3
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- Trial name
- NCT06421831
- Testing
- GC101
- Where
- China
- Volunteers
- 21 people
- Sponsor
- GeneCradle Inc
Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA)…
- Trial name
- SMA-REAL (NCT07737756)
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
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
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 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
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with…
- Trial name
- NCT01703988
- Testing
- Nusinersen
- Where
- 4 sites in United States
- Volunteers
- 34 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Neonatal Spinal Muscular Atrophy (SMA) Screening
Parents or legal guardian of neonates who signed agreement will receive SMA screening test if their neonates are affected with SMA.
- Trial name
- SMA (NCT03217578)
- Testing
- SMA Newborn Screening
- Where
- Taiwan
- Volunteers
- 250,000 people, healthy volunteers welcome
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Spinal Muscular Atrophy
Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System
In this project, we will establish the efficient and accurate gene dose determination system by combining the heterodulex analysis and gene dose analysis on DHPLC platform based on various quantitative and multiplex PCR…
- Trial name
- NCT00155168
- Where
- Taiwan
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital