Spinal Cord Stimulation for Spinal Muscular Atrophy

(SCSinSMA Trial)

Recruiting in Pittsburgh (>99 mi)

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

3 Participants Needed

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.

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 data supports the effectiveness of the treatment Spinal Cord Stimulator for Spinal Muscular Atrophy?

Research shows that spinal cord stimulation can improve motor control and reduce muscle stiffness in patients with upper motor neuron disorders and multiple sclerosis, suggesting it might help with similar symptoms in spinal muscular atrophy. ^{1 2 3 4 5}

Is spinal cord stimulation generally safe for humans?

Research shows that spinal cord stimulation (SCS) is generally safe for humans, with a low rate of serious adverse events. In a study of patients with chronic pain, the most common serious issue was implant site infection, occurring in less than 1% of cases. ^{6 7 8 9 10}

How does the treatment Spinal Cord Stimulator differ from other treatments for spinal muscular atrophy?

Spinal Cord Stimulation is unique because it involves using electrical impulses to improve motor control and reduce muscle stiffness, which is different from other treatments that may focus on genetic or drug-based approaches. This method has shown effectiveness in improving motor performance in other upper motor neuron disorders, suggesting potential benefits for spinal muscular atrophy. ^{1 11 12 13 14}

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.

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

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

Treatment Details

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.

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+

Findings from Research

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]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Neurophysiological evaluation of chronic spinal cord stimulation in patients with upper motor neuron disorders. [2019]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Chronic Spinal Cord Stimulation in the Treatment of Cerebral and Spinal Spasticity. [2019]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Spinal cord stimulation for multiple sclerosis: quantifiable benefits. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Fantastic Four: Age, Spinal Cord Stimulator Waveform, Pain Localization and History of Spine Surgery Influence the Odds of Successful Spinal Cord Stimulator Trial. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

A prospective, open-label, multicenter study to assess the efficacy of spinal cord stimulation and identify patients who would benefit. [2022]

6. New Zealand pubmed.ncbi.nlm.nih.gov

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

7. England pubmed.ncbi.nlm.nih.gov

Long-term safety of spinal cord stimulation systems in a prospective, global registry of patients with chronic pain. [2023]

8. United States pubmed.ncbi.nlm.nih.gov

Best Practices in Spinal Cord Stimulation. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

Magnetic resonance imaging in patients with spinal neurostimulation systems. [2007]

10. United States pubmed.ncbi.nlm.nih.gov

Are there a guidelines for implantable spinal cord stimulator therapy in patients using chronic anticoagulation therapy? - A review of decision-making in the high-risk patient. [2020]

11. United States pubmed.ncbi.nlm.nih.gov

Reliability of 4 outcome measures in pediatric spinal muscular atrophy. [2019]

12. Belgium pubmed.ncbi.nlm.nih.gov

Spinal motoneuron function in lower motor neuron disease: normal corticomotoneuronal and peripheral Ia EPSPs in patients with spinal muscular atrophy. [2015]

13. England pubmed.ncbi.nlm.nih.gov

Childhood spinal muscular atrophy: controversies and challenges. [2022]

14. United States pubmed.ncbi.nlm.nih.gov

Outcome measures for pediatric spinal muscular atrophy. [2019]