Spinal Cord Stimulation in Spinal Muscular Atrophy

Last updated: June 14, 2024
Sponsor: Marco Capogrosso
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Spinal Muscular Atrophy

Myasthenia Gravis (Chronic Weakness)

Muscular Dystrophy

Treatment

Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)

Clinical Study ID

NCT05430113
STUDY21080158
  • Ages 16-64
  • All Genders

Study Summary

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 3 or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 3 or 4 SMA aged 16 or older that show quantifiable motor deficits of the legs but are able to stand independently. The investigators will then implant the subjects with percutaneous, bilateral, linear spinal leads near the lumbar 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subject or subject's parent or legal guardian (for minor subjects) has providedwritten informed consent and Health Insurance Portability and Accountability Act (HIPAA) authorization, where applicable, prior to any study-related procedures.Minor subjects will be asked to give written assent according to local requirements.

  2. Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by determination ofa genetic deletion in the SMN1 gene (5q12.2-q13.3)

  3. Subject is diagnosed as having Type 3 or Type 4 SMA based on the following criteria

  4. Disease manifested after 18 months of age

  5. Disease manifested after ambulation was acquired

  6. Subject is ≥16 years of age and < 65 years of age

  7. Subject is able to stand independently for ≥3 seconds

  8. RHS score lower or equal to 65

  9. Subject (and subject's parent or legal guardian if subject is a minor) is willingand able to comply with scheduled visits and study procedures

Exclusion

Exclusion Criteria:

  1. Subject has deformation of the spinal canal preventing lead implantation as judgedby the study neurosurgeon

  2. Subject has size of spinal canal that is insufficient for lead implantation asjudged by the study neurosurgeon

  3. Subject has moderate or severe joint contractures that would affect ability toperform study measures

  4. Subject has severe behavioral or cognitive problems that preclude participation inthe study, in the opinion of the investigator

  5. Subject has previous or ongoing medical condition, medical history, physicalfindings or laboratory abnormalities that could affect safety of anesthesia or theprocedures, make it unlikely that intervention or follow-up will be correctlycompleted or impair the assessment of study results, in the opinion of theinvestigator

  6. Female subjects are pregnant or breastfeeding

  7. Subject has severe claustrophobia

  8. Subject is on anticoagulant, anti-spasticity or anti-seizure medication within 4weeks of lead implantation or requires these medications during the treatment phaseof the study

  9. Subject has medical implant that precludes magnetic resonance imaging

Study Design

Total Participants: 3
Treatment Group(s): 1
Primary Treatment: Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
Phase:
Study Start date:
April 05, 2022
Estimated Completion Date:
August 31, 2027

Study Description

The investigators plan to 1. verify that spinal cord stimulation increases hip muscle strength in subjects with SMA, 2. verify that spinal cord stimulation improves motor control in subjects with SMA, 3. verify that spinal cord stimulation induces measurable changes in spinal circuits and motoneuron recruitment properties in the 29 day course of implantation.

Connect with a study center

  • University of Pittsburgh

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

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