Clinical Trials Logo

Spinal Muscular Atrophy Type II clinical trials

View clinical trials related to Spinal Muscular Atrophy Type II.

Filter by:
  • None
  • Page 1

NCT ID: NCT06300996 Not yet recruiting - Clinical trials for Spinal Muscular Atrophy

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

Start date: September 2024
Phase: N/A
Study type: Interventional

Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with Type 2, 3, or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 2, 3, or 4 SMA aged 16 or older that show quantifiable motor deficits of the upper body. The investigators will then implant the subjects with percutaneous, linear spinal leads near the cervical spinal cord for a period of up to 29 days. Although these leads are not optimized for motor function but rather for their clinically approved indication of treating pain, the investigators believe they provide a safe technology enabling our team to perform scientific measurement necessary to evaluate potential for effects of SCS in motor paralysis with SMA. After the end of the study, the leads will be explanted.

NCT ID: NCT06137612 Enrolling by invitation - Clinical trials for Spinal Muscular Atrophy Type II

Spinal Cord Gray Matter Imaging in Spinal Muscular Atrophy

Start date: August 17, 2020
Phase:
Study type: Observational

This study aims to measure the spinal cord gray matter in patients with spinal muscular atrophy (SMA) type II and III in comparison with age- and sex-matched healthy controls (HC) using rAMIRA (radially sampled averaged magnetization inversion recovery acquisitions) imaging, a novel MRI (Magnetic Resonance Imaging) method. Patient and HC undergo MRI examinations, clinical/neurological (handheld dynamometry) and electrophysiological investigations (MUNIX, Motor Unit Number Index). Serum markers of neuro-axonal and astrocytic injury are also assessed.

NCT ID: NCT05715749 Active, not recruiting - Clinical trials for Spinal Muscular Atrophy Type II

Body Weight Support Harness System in Spinal Muscular Atrophy

Start date: September 7, 2018
Phase: N/A
Study type: Interventional

The goal of this pilot interventional study is to learn about the use of an in-home harness system in children who have been treated for spinal muscular atrophy. The main questions it aims to answer are: 1. Is the in-home body weight support harness system a feasible option for families to use? 2. Is the in-home body weight support harness system a useful tool for children treated for spinal muscular atrophy? 3. Is the in-home body weight support harness system a safe tool for children treated for spinal muscular atrophy? Participants will be given an in-home body weight support harness system and taught how to use it. Families will document how often and for how long they use the system over 6 months. Children will be given tests of motor function at the beginning, 3-months, and 6-months. At the end of the study, families will be asked to fill out a questionnaire about thier experience using the system.

NCT ID: NCT05626855 Active, not recruiting - Clinical trials for Neuromuscular Diseases

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX

ONYX
Start date: April 17, 2023
Phase: Phase 3
Study type: Interventional

The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.

NCT ID: NCT05416034 Completed - Clinical trials for Spinal Muscular Atrophy Type II

Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular Atrophy

Start date: December 1, 2017
Phase: N/A
Study type: Interventional

The purpose of this study is to evaluate the impact of the use of a pediatric exoskeleton on the quality of life of children, specifically in the psychological and care dimensions. Other objectives are to evaluate changes at the physical and functional level.

NCT ID: NCT04042025 Active, not recruiting - SMA Clinical Trials

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Start date: February 10, 2020
Phase: Phase 3
Study type: Interventional

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

NCT ID: NCT03709784 Recruiting - Clinical trials for Spinal Muscular Atrophy

Spinraza in Adult Spinal Muscular Atrophy

SAS
Start date: August 16, 2018
Phase:
Study type: Observational

This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.

NCT ID: NCT01302600 Completed - Clinical trials for Spinal Muscular Atrophy Type II

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.

Start date: November 2010
Phase: Phase 2
Study type: Interventional

Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years

NCT ID: NCT00439569 Terminated - Clinical trials for Spinal Muscular Atrophy Type II

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NPTUNE01
Start date: January 2008
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.