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

Launched by MARCO CAPOGROSSO · Mar 1, 2024

Keywords

ClinConnect Summary

This clinical trial is exploring whether spinal cord stimulation (SCS) can help improve movement in people with spinal muscular atrophy (SMA), specifically those with types 2, 3, or 4. SCS is a technique that has previously shown promise in helping people with spinal cord injuries regain some motor function. The study will enroll up to six participants aged 16 to 64 who have measurable difficulties using their upper body. To qualify, participants must have a confirmed diagnosis of SMA and be non-ambulatory, meaning they cannot walk independently. Additionally, they should have been receiving specific therapies for SMA for at least six months.

Participants in the trial will have small electrodes implanted near their cervical spinal cord for up to 29 days. These electrodes are usually used for treating pain but will be studied here to assess whether they can also help with motor function in SMA. At the end of the study, the electrodes will be removed. It’s important to note that participants will need to attend scheduled visits and follow study procedures. The trial is currently recruiting participants, and both patients and healthy individuals can apply, provided they meet specific criteria.

Gender

ALL

Eligibility criteria

• SMA Participant Inclusion Criteria:
• • 1. Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by determination of a genetic deletion in the SMN1 gene (5q12.2-q13.3).
• 2. Subject is diagnosed as being non-ambulatory SMA based on the following criteria:
• • a. Can't stand independently.
• • 3. Subject is ≥16 years of age and \< 65 years of age.
• • 4. Subject is able to sit independently.
• • 5. A minimum score of 1 for Entry Item "A" of the Revised Upper Limb Module (RULM) scale for SMA: "Can use hands to hold pencil or pick up a coin/token or drive a powered chair, use phone key pad"
• • 6. Subject (and subject's parent or legal guardian if subject is a minor) is willing and able to comply with scheduled visits and study procedures
• • 7. Participants must have started SMN inducing therapies (Spinraza or risdisplam) at least 6 months prior to enrollment. (They must have either gotten their first injection at at least 6 months prior, or they started daily intake of risdisplam at least 6 months prior to the study)
• Healthy Control Participant Inclusion Criteria:
• • 1. Subject is ≥18 years of age and \< 65 years of age.
• • 2. Subject is able to stand independently for ≥3 seconds.
• • 3. Subject is willing and able to comply with scheduled visits and study procedures.
• SMA Participant Exclusion Criteria:
• • 1. Subject has deformation of the spinal canal preventing lead implantation as judged by the study neurosurgeon
• • 2. Subject has size of spinal canal that is insufficient for lead implantation as judged by the study neurosurgeon
• • 3. Subject has moderate or severe joint contractures that would affect ability to perform study measures, determined by the study physician
• • 4. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator
• • 5. Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety of anesthesia or the procedures, make it unlikely that intervention or follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator
• • 6. Female subjects are pregnant or breastfeeding, as established by self-report.
• • 7. Subject has severe claustrophobia
• • 8. Subject is on anticoagulant, anti-spasticity or anti-seizure medication within 4 weeks of lead implantation or requires these medications during the treatment phase of the study
• • 9. Subject has medical implant that precludes magnetic resonance imaging
• • 10. Subject has a deconditioned respiratory system, per the discretion of the physician investigator.
• • 11. Subjects with renal insufficiency at the discretion of the physician investigator.
• • 12. Subjects requiring any form of sedation for MRI will be excluded.
• Healthy Control Participant Exclusion Criteria:
• • 1. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator would impact participation in the study.
• • 2. Participants who have any serious disease or disorder (e.g. cancer, severe cardiac or respiratory disease, neurological conditions other than stroke, etc.) or cognitive impairments that could affect their ability to participate in this study.
• • 3. Female subjects are pregnant or breastfeeding, as established by self-report.
• • 4. Subjects requiring sedation for MRI will be excluded.