What is the mechanism of action of this treatment?

Spinal Cord Stimulation (SCS) for Spinal Muscular Atrophy (SMA) works by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical inputs. This mechanism is crucial for SMA patients as it aims to improve motor function by making the remaining neural pathways more effective in transmitting signals. This can potentially lead to better muscle control and strength, which are often compromised in SMA. Enhancing neural circuit responsiveness is particularly important for maintaining and improving the quality of life in SMA patients, as it can help mitigate some of the motor deficits associated with the disease.

What side effects are associated with this type of intervention?

Spinal cord stimulation (SCS) for Spinal Muscular Atrophy (SMA) primarily involves the implantation of leads near the lumbar spinal cord. Common side effects of SCS include discomfort or pain at the implantation site, infection, lead migration, and hardware malfunction. Additionally, patients may experience muscle spasms, tingling, or numbness. These side effects are generally mild and manageable, but serious complications, although rare, can occur.

What prior FDA approvals exist?

The FDA has approved several treatments for Spinal Muscular Atrophy (SMA), including nusinersen (Spinraza), onasemnogene abeparvovec-xioi (Zolgensma), and risdiplam (Evrysdi). These treatments primarily focus on genetic and molecular approaches to increase the production of the survival motor neuron (SMN) protein, which is deficient in SMA patients. Unlike these pharmacological treatments, the trial on spinal cord stimulation aims to enhance motor function by recruiting afferent input to improve the responsiveness of neural circuits. This neuromodulation approach is distinct from the genetic therapies currently approved by the FDA.

What other types of research exist?

Promising novel alternatives to Spinal Cord Stimulation for Spinal Muscular Atrophy patients include transcutaneous supraorbital nerve stimulation and remote electrical neuromodulation. These techniques have shown efficacy in reducing pain and improving neural responsiveness in other conditions and may offer potential benefits for SMA patients.

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Spinal Cord Stimulation

Spinal Cord Stimulation for Spinal Muscular Atrophy (SCSinSMA Trial)

N/A

Waitlist Available

Research Sponsored by Marco Capogrosso

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by determination of a genetic deletion in the SMN1 gene (5q12.2-q13.3)

Be younger than 65 years old

Must not have

Female subjects are pregnant or breastfeeding

Subject has moderate or severe joint contractures that would affect ability to perform study measures

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 29 days

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects may include discomfort at the implant site, infection risk from surgery, possible nerve damage due to lead placement near the spine, and general risks associated with anesthesia during implantation and explantation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently pregnant or breastfeeding.

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I have severe joint stiffness that limits my movement.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 29 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~29 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 29 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Muscle Weakness

Number and Severity of Adverse Events

Secondary study objectives

Cortico-spinal Tract Integrity

Discomfort/Pain

Impression

+11 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Spinal Cord StimulationExperimental Treatment1 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.

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Spinal Cord Stimulation (SCS) for Spinal Muscular Atrophy (SMA) works by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical inputs. This mechanism is crucial for SMA patients as it aims to improve motor function by making the remaining neural pathways more effective in transmitting signals. This can potentially lead to better muscle control and strength, which are often compromised in SMA. Enhancing neural circuit responsiveness is particularly important for maintaining and improving the quality of life in SMA patients, as it can help mitigate some of the motor deficits associated with the disease.

Vibratory stimulation. Part III. Possible applications of vibration in treatment of motor dysfunctions.Transcranial Static Magnetic Field Stimulation of the Motor Cortex in Children.Effectiveness of neuromuscular electrical stimulation for management of shoulder subluxation post-stroke: a systematic review with meta-analysis.