Spinal Muscular Atrophy Type 3SCSinSMANCT05430113
Spinal Cord Stimulation in Spinal Muscular Atrophy
A study testing Spinal Cord Stimulator (octopolar Medtronic Vectris Leads) in people with Spinal Muscular Atrophy Type 3 and Spinal Muscular Atrophy Type 4, run by Marco Capogrosso at 1 site in United States.
Trial 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 circuits to residual cortical inputs. Read more about this study
- Who is it for?
- Ages 16 to 64 years
- Conditions the study is for
- Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
- What is being tested?
- Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Apr 2022to 13 Jan 2025
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 9Show fewer
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
About this study
Official title: Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy
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 circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with type 3 or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 3 or 4 SMA aged 16 or older that show quantifiable motor deficits of the legs but are able to stand independently. The investigators will then implant the subjects with percutaneous, bilateral, linear spinal leads near the lumbar spinal cord for a period of up to 29 days. Although these leads are not optimized for motor function but rather for their clinically approved indication of treating pain, the investigators believe they provide a safe technology enabling our team to perform scientific measurement necessary to evaluate potential for effects of SCS in motor paralysis with SMA. After the end of the study, the leads will be explanted.
Read the 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, 3. verify that spinal cord stimulation induces measurable changes in spinal circuits and motoneuron recruitment properties in the 29 day course of implantation.
- Being tested
- Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
- Keywords
- Spinal Muscular Atrophy, Spinal Cord Stimulation
- Medical subject headings
- Spinal Muscular Atrophies of Childhood, Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)Device
2-4 leads FDA-approved for treatment of symptoms of refractory pain
| Study group | Role | What they receive |
|---|---|---|
| Spinal Cord Stimulation | Experimental | 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. Receives: Device: Spinal Cord Stimulator (octopolar Medtronic Vectris Leads) |
| Control Arm | No intervention | Healthy volunteers may be enrolled for comparison data for those who have received the spinal cord leads. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Muscle Weakness
Isometric torque: measure the isometric torque produced by the subject at the hip during hip-flexion. Comparison of SCS-on with SCS-off performance. Success Criteria: ≥20% increased torque production over SCS-off baseline as measured during single-joint isometric torque.
Measured: 29 days
Number of Participants With Adverse Events
Success Criteria: no serious adverse event related to the stimulation or intolerable adverse event reported
Measured: 29 days
Secondary outcomes (14)
Motor Function ROM
Range of Motion (ROM): Meaningful Change: increase of >20% of the hip joint (if available) and the knee joint (if available) during SCS against SCS-off as measured by the HUMAC NORM during single-joint isotonic trials.
Measured: 29 days
Muscle Weakness 2
Muscle activation: measure surface EMGs produced by the subjects during isometric movements of the hip, knee, and ankle in the HUMAC Norm and compare SCS-on with SCS-off performances. Meaningful Change:<20% EMG RMS compared to SCS-on.
Measured: 29 days
Motor Function: 6-Minute Walk Test
6 minute walk test: perform the 6-minute walk test with SCS-on and SCS-off and distance between SCS-on and SCS-off is set to 24 m. If not ambulatory, any increased ambulation distance from SCS-off condition.
Measured: 29 days
Motor Function: Fatigue
Fatigue will be assessed during motor function tests.
Measured: 29 days
Motor Function RHS
The Revised Hammersmith Functional Scale is a further refined version of the Hammersmith Functional Motor Scale Extended and includes specific items focused on lower-limb motor control such as hip-flexion, standing and walking a that are particularly relevant for the type III population of study. The scale is a 36 item performance evaluation, with a score range from 0-69 in which higher scores indicate better performance.
Meaningful Change: ≥2 point improvement. Compare outcomes between SCS-on and SCS-off. Motor Function Measure 32. The 32 items MFM is often used in trials of neuromuscular disorders, hence it provides a meaningful comparison against outcomes of other trials. Meaningful Change: ≥2 point improvement. Compare outcomes between SCS-on and SCS-off.
Measured: 29 days
Number of Participants With Discomfort/Pain
Patients will be asked to provide a score from 1 to 10 where a greater number indicates a greater amount of discomfort for each stimulation configuration. Spinal cord stimulation produces tingling sensations and other type of sensory phenomena. It is important to document that stimulation intensities required to improve motor function remain within a range of non-painful sensations.
Measured: 29 days
Sensorimotor Network Structure Density
The investigators will perform High-definition Diffusion Weighted Imaging to quantify Fractional Anisotropy as a measurement of axon density in the brain and spinal cord pre and post study.
Measured: 29 days
Impression
The investigators will collect subjects and therapist feedback on how the technology is performing and what they would want to modify using The Clinical Global Impression Scale, a scale of 1-7 where a lower number indicates better performance and a higher number indicates more greatly impacted by their disease.
Measured: 29 days
Sensorimotor Network Structure Integrity
The investigators will perform High-definition Diffusion Weighted Imaging to quantify Fractional Anisotropy as a measurement of axon integrity in the brain and spinal cord pre and post study.
Measured: 29 days
Sensorimotor Network Function
The investigators will perform resting state and motor-task functional MRI of the brain and spinal cord to quantify neural network activation at rest and during the execution of simple motor task such as leg muscle contraction.
Measured: 29 days
Cortico-spinal Tract Integrity
The investigators will measure muscle evoked potential consequent to Transcranial Magnetic Stimulation of the cortico-spinal tract to assess integrity of the cortico-spinal tract.
Measured: 29 days
Spinal Circuit Excitability
The investigators will measure H-reflexes of leg muscles to quantify excitability of spinal motoneurons to stimulation of primary sensory afferents pre and post-study. Expected Result: Our main scientific hypothesis is that SCS will restore monosynaptic responses of weak spinal motoneurons, thus increasing H-reflex responses pre and post-study.
Measured: 29 days
Motoneuron Firing Rates
The investigators will use high-density EMGs on leg muscles to calculate firing rates of single spinal motoneuron discharge during isometric maximal voluntary contractions.
Measured: 29 days
Motor Firing Number
The investigators will use high-density EMGs on leg muscles to calculate the number of firing rates of single spinal motoneuron discharge during isometric maximal voluntary contractions.
Measured: 29 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2022
- Study start5 Apr 2022
- Primary completion22 Jan 2024
- Study completion13 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular Atrophy Type 3Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Single-arm, open-label study performed to quantify variables that are predictive of the efficacy of spinal cord stimulation to improve motor control.
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marco Capogrosso
- Sponsor type
- University, hospital or other
- Collaborators
- Roche-Genentech
- Responsible party
- Sponsor-investigator, Marco Capogrosso, University of Pittsburgh
- Investigators
- Marco Capogrosso, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05430113
- Sponsor's protocol ID
- STUDY21080158
- Acronym
- SCSinSMA
- Status last verified
- Jan 2026
- First submitted
- 12 Apr 2022
- Last updated
- 16 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: Data may be shared with other researchers for the purpose of data analysis and collaboration.
Publications
Prat-Ortega G, Ensel S, Donadio S, Borda L, Boos A, Yadav P, Verma N, Ho J, Carranza E, Frazier-Kim S, Fields DP, Fisher LE, Weber DJ, Balzer J, Duong T, Weinstein SD, Eliasson MJL, Montes J, Chen KS, Clemens PR, Gerszten P, Mentis GZ, Pirondini E, Friedlander RM, Capogrosso M. First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy. Nat Med. 2025 Apr;31(4):1246-1256. doi: 10.1038/s41591-024-03484-8. Epub 2025 Feb 5.
PubMed 39910271
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT05430113
- Acronym / sponsor ID
- SCSinSMA / STUDY21080158
- Keywords
- Spinal Muscular Atrophy, Spinal Cord Stimulation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Marco CapogrossoUniversity, hospital or other
- Collaborators
- Roche-Genentech
- Study start
- 5 Apr 2022 (actual)
- Primary completion
- 22 Jan 2024 (actual)
- Study completion
- 13 Jan 2025 (actual)
- First posted
- 24 Jun 2022 (actual)
- Results
- Posted 16 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with type 3 or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 3 or 4 SMA aged 16 or older that show quantifiable motor deficits of the legs but are able to stand independently. The investigators will then implant the subjects with percutaneous, bilateral, linear spinal leads near the lumbar spinal cord for a period of up to 29 days. Although these leads are not optimized for motor function but rather for their clinically approved indication of treating pain, the investigators believe they provide a safe technology enabling our team to perform scientific measurement necessary to evaluate potential for effects of SCS in motor paralysis with SMA. After the end of the study, the leads will be explanted.
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, 3. verify that spinal cord stimulation induces measurable changes in spinal circuits and motoneuron recruitment properties in the 29 day course of implantation.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Single-arm, open-label study performed to quantify variables that are predictive of the efficacy of spinal cord stimulation to improve motor control.
| Arm | Type | Receives |
|---|---|---|
| Spinal Cord Stimulation | Experimental | Device: Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)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. |
| Control Arm | No intervention | –Healthy volunteers may be enrolled for comparison data for those who have received the spinal cord leads. |
Who may qualify
- Ages
- 16 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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
- Subject is ≥16 years of age and < 65 years of age
- Subject is able to stand independently for ≥3 seconds
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)2-4 leads FDA-approved for treatment of symptoms of refractory pain | Device | Spinal Cord Stimulation |
Primary outcome
Muscle Weakness
Time frame: 29 days
Isometric torque: measure the isometric torque produced by the subject at the hip during hip-flexion. Comparison of SCS-on with SCS-off performance. Success Criteria: ≥20% increased torque production over SCS-off baseline as measured during single-joint isometric torque.
Number of Participants With Adverse Events
Time frame: 29 days
Success Criteria: no serious adverse event related to the stimulation or intolerable adverse event reported
14 secondary outcomes
Motor Function ROM
Time frame: 29 days
Muscle Weakness 2
Time frame: 29 days
Motor Function: 6-Minute Walk Test
Time frame: 29 days
Motor Function: Fatigue
Time frame: 29 days
Motor Function RHS
Time frame: 29 days
Number of Participants With Discomfort/Pain
Time frame: 29 days
Sensorimotor Network Structure Density
Time frame: 29 days
Impression
Time frame: 29 days
Sensorimotor Network Structure Integrity
Time frame: 29 days
Sensorimotor Network Function
Time frame: 29 days
Cortico-spinal Tract Integrity
Time frame: 29 days
Spinal Circuit Excitability
Time frame: 29 days
Motoneuron Firing Rates
Time frame: 29 days
Motor Firing Number
Time frame: 29 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39910271Prat-Ortega G, Ensel S, Donadio S, Borda L, Boos A, Yadav P, Verma N, Ho J, Carranza E, Frazier-Kim S, Fields DP, Fisher LE, Weber DJ, Balzer J, Duong T, Weinstein SD, Eliasson MJL, Montes J, Chen KS, Clemens PR, Gerszten P, Mentis GZ, Pirondini E, Friedlander RM, Capogrosso M. First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy. Nat Med. 2025 Apr;31(4):1246-1256. doi: 10.1038/s41591-024-03484-8. Epub 2025 Feb 5.
- Individual participant dataSharing planned: Yes. Data may be shared with other researchers for the purpose of data analysis and collaboration.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Marco Capogrosso principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Spinal Cord Stimulator (octopolar Medtronic Vectris Leads) in people with Spinal Muscular Atrophy Type 3 and Spinal Muscular Atrophy Type 4, run by Marco Capogrosso at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Muscle Weakness (time frame: 29 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05430113, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2026.