Spinal Muscular Atrophy Clinical Trials
A listing of Spinal Muscular Atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 67 clinical trials
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) (STEER)
To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age over a 15
- 4 views
- 29 Jul, 2023
- 27 locations
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.
- 0 views
- 20 Sep, 2023
- 1 location
The DISSECT Study: Effect of Peri-aDventItial SMA diSsECtion on Margin sTatus During Pancreaticoduodenectomy for Resectable Pancreatic Cancer
There is a high rate of R1 resection following patients undergoing pancreaticoduodenectomy for pancreatic cancer. The most commonly positive margin is the SMA. Peri-adventitial dissection has
- 1 views
- 04 Jun, 2021
- 1 location
Neurofeedback From the Supplementary Motor Area for Tourette Syndrome
This is a clinical trial where adolescents aged 10-16 years old with Tourette Syndrome (or chronic tic disorder) are randomized to receive either real-time functional magnetic resonance imaging (fMRI) neurofeedback targeting the supplementary motor area (for the experimental intervention) or real-time fMRI neurofeedback (NF) from a control region (for the …
- 0 views
- 06 Apr, 2023
- 1 location
Development of Non-Invasive Brain Stimulation Techniques
Background Noninvasive brain stimulation (NIBS) may help diagnose and treat psychiatric and neurological illness. But there is not enough research on how to apply NIBS. This includes how strong to make it, where on the brain to apply it, and for how long. Researchers also want to see what the …
- 128 views
- 17 Sep, 2023
- 1 location
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
- 0 views
- 08 Sep, 2023
- 2 locations
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam.
- 0 views
- 04 Apr, 2023
- 13 locations
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy (RESILIENT)
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.
- 0 views
- 16 Sep, 2023
- 38 locations
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE)
safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either
- 0 views
- 07 Sep, 2023
- 13 locations
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (SAPPHIRE)
This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and
- 0 views
- 12 Aug, 2023
- 45 locations