Spinal Cord Stimulation for Spinal Muscular Atrophy

(SCSinSMA Trial)

Overseen By Sydney Bader, MS

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Marco Capogrosso

Must not be taking: Anticoagulants, Anti-spasticity, Anti-seizure

Disqualifiers: Spinal deformation, Joint contractures, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 29

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether spinal cord stimulation (SCS) can improve movement in individuals with spinal muscular atrophy (SMA), specifically types 3 and 4. SCS uses electrical signals to activate nerves and may enhance movement in people with SMA, although it is typically used for pain relief. The trial involves temporarily implanting a spinal cord stimulator device near the spinal cord for up to 29 days. Suitable candidates have experienced SMA symptoms after learning to walk and can currently stand unassisted for at least 3 seconds. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could enhance mobility for those with SMA.

Will I have to stop taking my current medications?

Yes, you will need to stop taking anticoagulant, anti-spasticity, or anti-seizure medications at least 4 weeks before the lead implantation and during the treatment phase of the study.

What prior data suggests that spinal cord stimulation is safe for spinal muscular atrophy?

Research has shown that spinal cord stimulation (SCS) is generally safe for people. One study found that SCS can improve muscle strength in individuals with spinal muscular atrophy (SMA), indicating the treatment is both potentially effective and well-tolerated. Another report highlighted that SCS significantly reduces pain in patients with various conditions, suggesting it is a safe technology.

The FDA has already approved the equipment used in the trial for managing pain, which boosts confidence in its safety. While any medical procedure carries some risks, existing data supports that SCS is generally safe. Participants in similar studies have experienced positive results with manageable side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Spinal cord stimulation is unique because it offers a new way to manage spinal muscular atrophy (SMA) by directly targeting the nervous system. Unlike traditional treatments that primarily focus on slowing disease progression or managing symptoms through medication, this method involves implanting a device that sends electrical impulses to the spinal cord, potentially improving motor function and quality of life. Researchers are excited because this approach could provide a novel and effective option for those with SMA, offering hope for enhanced mobility and independence.

What evidence suggests that spinal cord stimulation is effective for spinal muscular atrophy?

Research has shown that spinal cord stimulation (SCS) can improve movement in people with spinal cord injuries by enhancing the activity of functioning nerve pathways. In studies involving individuals with spinal muscular atrophy (SMA), SCS has demonstrated potential in increasing muscle strength and improving movement. Early results from one study indicated that SCS improved strength, endurance, and walking ability in adults with SMA. In this trial, all participants will receive spinal cord stimulation to further evaluate its effectiveness in addressing movement difficulties in people with SMA. Although more research is needed, the initial results are promising. ^{1 2 4 5 6}

Who Is on the Research Team?

Marco Capogrosso

Principal Investigator

University of Pittsburgh

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive spinal cord stimulation with implanted leads for up to 29 days to assess improvements in motor deficits

4 weeks

Continuous monitoring during the 29-day implantation period

Follow-up

Participants are monitored for safety and effectiveness after treatment, including explantation of leads

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Spinal Cord Stimulator

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

In a study of 69 patients with implanted spinal cord stimulators (SCSs) who underwent 78 MRI scans, the overall adverse event rate for off-label MRI use was low at 9.72%, with no serious adverse events reported.

All reported clinical adverse events were minor and resolved, suggesting that the benefits of MRI for pain management in patients with older SCS models may outweigh the associated risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Off-Label Magnetic Resonance Imaging (MRI) in Patients with Persistent Pain with Spinal Cord Stimulators: A Case Series. Ragukonis, T.[2022]

In a global study involving 1881 participants, spinal cord stimulation (SCS) demonstrated a high level of safety, with a low annualized device explant rate of 3.5% and only 1.1% due to inadequate pain relief.

The most common serious adverse event was implant site infection, occurring in less than 1% of cases, indicating that SCS is generally safe for chronic pain management over a follow-up period of up to 3 years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety of spinal cord stimulation systems in a prospective, global registry of patients with chronic pain. Rauck, RL., Loudermilk, E., Thomson, SJ., et al.[2023]

A study involving 31 patients with spinal cord stimulation (SCS) systems showed that undergoing MRI scans is generally safe, with no serious complications or harm reported during the procedure.

While some minor issues like increased temperature at the generator site and telemetry difficulties were noted, these did not affect patient safety or lead to reprogramming of the SCS systems, indicating that MRI can be performed safely in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Magnetic resonance imaging in patients with spinal neurostimulation systems. De Andres, J., Valía, JC., Cerda-Olmedo, G., et al.[2007]

Citations

1. rnel.pitt.edu rnel.pitt.edu/scs-sma

Spinal Cord Stimulation in Spinal Muscular Atrophy We aim to test if Spinal Cord Stimulation (SCS) is a safe and effective way to improve motor function in people living with Spinal Muscular Atrophy (SMA).

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9966619/

First Use of Non-Invasive Spinal Cord Stimulation in Motor ... Spinal cord stimulation may be an effective rehabilitation method in patients treated with nusinersen. More research is needed. Keywords: spinal ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06300996?spons=Roche&viewType=Table&rank=7

Spinal Cord Stimulation for the Treatment of Motor Deficits ... Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to ...

4. curesma.org curesma.org/wp-content/uploads/2025/03/Prat-Ortega-Epidural-spinal-cord-stimulation-in-SMA-Nature-Medicine-2025.pdf

First-in-human study of epidural spinal cord stimulation in ... Here we show that epidural spinal cord stimulation (SCS) improved motoneuron function, thereby increasing strength, endurance and gait quality, in three adults ...

5. upmc.com upmc.com/media/news/020525-spinal-cord-stimulation

Spinal Cord Stimulation Restores Neural Function, Targets ... Spinal cord stimulation improves muscle strength in individuals with spinal muscle atrophy.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12321793/

The effectiveness of spinal cord stimulation combined with ... Spinal cord stimulation alone led to significant pain reductions, with responder rates above 80% and average decreases of 5–6 cm on pain scales.