Status:
ACTIVE_NOT_RECRUITING
Spinal Cord Stimulation in Spinal Muscular Atrophy
Lead Sponsor:
Marco Capogrosso
Collaborating Sponsors:
Roche-Genentech
Conditions:
Spinal Muscular Atrophy Type 3
Spinal Muscular Atrophy Type 4
Eligibility:
All Genders
16-64 years
Phase:
NA
Brief 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 ci...
Detailed 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,...
Eligibility Criteria
Inclusion
- 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.
- Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by determination of a genetic deletion in the SMN1 gene (5q12.2-q13.3)
- Subject is diagnosed as having Type 3 or Type 4 SMA based on the following criteria
- Disease manifested after 18 months of age
- Disease manifested after ambulation was acquired
- Subject is ≥16 years of age and \< 65 years of age
- Subject is able to stand independently for ≥3 seconds
- RHS score lower or equal to 65
- 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
- Subject has deformation of the spinal canal preventing lead implantation as judged by the study neurosurgeon
- Subject has size of spinal canal that is insufficient for lead implantation as judged by the study neurosurgeon
- Subject has moderate or severe joint contractures that would affect ability to perform study measures
- Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator
- 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
- Female subjects are pregnant or breastfeeding
- Subject has severe claustrophobia
- 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
- Subject has medical implant that precludes magnetic resonance imaging
Key Trial Info
Start Date :
April 5 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2027
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT05430113
Start Date
April 5 2022
End Date
August 1 2027
Last Update
May 16 2025
Active Locations (1)
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1
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213