Study Summary
Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in a variety of conditions. This pilot study will test if SCS can show evidence in improving motor deficits in people with Type 3 or Type 4 Spinal Muscular Atrophy (SMA). Willing participants with Type 3 or Type 4 SMA who are 16 years or older will be implanted for 29 days to help quantify if SCS shows improvement in motor deficits.
Inclusion Criteria:
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Subject or subject’s parent or legal guardian (for minor subjects) has provided written 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.
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Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by determination of a genetic deletion in the SMN1 gene (5q12.2-q13.3)
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Subject is diagnosed as having Type 3 or Type 4 SMA based on the following criteria
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Disease manifested after 18 months of age
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Disease manifested after ambulation was acquired
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Subject is ≥16 years of age and < 65 years of age
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Subject is able to stand independently for ≥3 seconds
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RHS score lower or equal to 65
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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
Exclusion Criteria:
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Subject has deformation of the spinal canal preventing lead implantation as judged by the study neurosurgeon
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Subject has size of spinal canal that is insufficient for lead implantation as judged by the study neurosurgeon
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Subject has moderate or severe joint contractures that would affect ability to perform study measures
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Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator
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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
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Female subjects are pregnant or breastfeeding
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Subject has severe claustrophobia
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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
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Subject has medical implant that precludes magnetic resonance imaging
Principle Investigator:
Marco Capogrosso, PhD
For more infomation, please contact:
Sydney Bader via email (syb17@pitt.edu) or phone (412-648-4196)
