Spinal Cord Stimulation for Spinal Muscular Atrophy

(SCSinSMA Trial)

Overseen by Marco Capogrosso

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Marco Capogrosso

No Placebo Group

Trial Summary

What is the purpose of this trial?

This trial tests if spinal cord stimulation (SCS) can improve leg movement in people with Type 3 or 4 Spinal Muscular Atrophy (SMA) who can stand but have motor deficits. SCS uses electrical pulses to help the spinal cord communicate better with the brain, potentially improving muscle control and strength. Spinal cord stimulation (SCS) has been shown to improve motor performance in various conditions, including certain types of muscle weakness.

Research Team

Marco Capogrosso

Principal Investigator

University of Pittsburgh

Eligibility Criteria

This trial is for individuals aged 16-65 with Type 3 or 4 spinal muscular atrophy (SMA), confirmed by a genetic test. Participants must be able to stand independently and have a specific RHS score. They should not have severe joint contractures, claustrophobia, spinal deformations, cognitive issues that affect study participation, or conditions affecting safety of procedures.

Inclusion Criteria

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.

You have a specific kind of spinal muscular atrophy (SMA) that started after you were 18 months old or after you learned to walk. You are between 16 and 65 years old, can stand independently for at least 3 seconds, have a certain score on a test, and are willing to attend study visits and follow study instructions.

I have been diagnosed with 5q-SMA based on a genetic test.

Exclusion Criteria

You have a condition in your spine that makes it impossible to implant the lead, as decided by the study neurosurgeon.

I am currently pregnant or breastfeeding.

Your spinal canal is not big enough for the study neurosurgeon to put in the lead.

Treatment Details

Interventions

• Spinal Cord Stimulator (Spinal Cord Stimulation)

Trial Overview The trial tests if Spinal Cord Stimulation (SCS) using octopolar Medtronic Vectris Leads can improve motor function in SMA patients. Up to six subjects will receive temporary lumbar spine implants for up to 29 days to evaluate the potential effects on motor paralysis.

Participant Groups

Group I: Spinal Cord Stimulation Experimental Treatment 1 Intervention

All patients will receive FDA-approved percutaneous spinal cord stimulation leads implanted in the epidural (T12-L2 vertebra) space. The leads will be connected to external stimulators (either FDA-approved or human-grade research stimulator with safety features) during research activities.

Who Is Running the Clinical Trial?

Marco Capogrosso

Lead Sponsor

Trials

3

Recruited

40+

Roche-Genentech

Industry Sponsor

Trials

27

Recruited

3,800+