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.

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Found 79 clinical trials
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE)

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured

muscle atrophy
gene deletion
  • 11 Jul, 2022
  • 53 locations
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

  • 25 Jul, 2022
  • 4 locations
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

The study is a randomized, single oral dose, crossover study in up to three parts to investigate the relative bioavailability and bioequivalence of two different formulations of risdiplam 5 mg (dispersible tablets) versus the current risdiplam oral solution formulation in healthy male and female participants. The effect of food on …

Accepts healthy volunteers
  • 29 Jul, 2022
  • 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

  • 04 Jun, 2021
  • 1 location
The Effect of Repetitive Transcranial Magnetic Stimulation on the Cognition in People With Parkinson's Disease

) mode at supplementary motor area (SMA). Cognitive bahevorial tests and other motor/depression assessments will be assessed before and after the intervention.

  • 13 Apr, 2021
  • 1 location
Nivolumab, Ipilimumab and Chemoradiation in Treating Patients With Locally Advanced Pancreatic Cancer. (LAPTOP)

advanced pancreatic cancer (LAPC)) is present in 30% of the cases and is defined as a surgically unresectable tumor encasing the adjacent arteries [celiac axis, superior mesenteric artery (SMA)]. In these

  • 08 Aug, 2022
  • 1 location
Repetitive Transcranial Magnetic Stimulation on Freezing of Gait in People With Parkinson's Disease

. All the study subjects will receive rTMS under intermittent theta burst stimulation (iTBS) mode at supplementary motor area (SMA). Gait analysis and other motor performance will be assessed before and

  • 11 Apr, 2021
  • 1 location
Outcomes of Ablation of Unresectable Pancreatic Cancer Using the NanoKnife Irreversible Electroporation (IRE) System

The purpose of this study is to evaluate the short and intermediate term outcomes of the NanoKnife Irreversible Electroporation System when used to treat unresectable pancreatic cancer. In addition, the study will evaluate the efficacy of this device in treating symptoms of unresectable pancreatic cancer. The NanoKnife, System has been …

  • 08 Apr, 2022
  • 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 …

Accepts healthy volunteers
  • 24 Jul, 2022
  • 1 location
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)

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

a 12
  • 04 Jun, 2022
  • 11 locations