Spinal Muscular AtrophyNCT06300996
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb
A study testing Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads) in people with Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, 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 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II
- 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
- 31 May 2024to 19 Oct 2026
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
SMA Participant Inclusion Criteria:
- 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 being non-ambulatory SMA based on the following criteria:
- Can't stand independently.
- Subject is ≥16 years of age and < 65 years of age.
- Subject is able to sit independently.
- A minimum score of 1 for Entry Item "A" of the Revised Upper Limb Module (RULM) scale for SMA: "Can use hands to hold pencil or pick up a coin/token or drive a powered chair, use phone key pad"
- 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
- Participants must have started SMN inducing therapies (Spinraza or risdisplam) at least 6 months prior to enrollment. (They must have either gotten their first injection at at least 6 months prior, or they started daily intake of risdisplam at least 6 months prior to the study)
Healthy Control Participant Inclusion Criteria:
- Subject is ≥18 years of age and < 65 years of age.
- Subject is able to stand independently for ≥3 seconds.
- Subject is willing and able to comply with scheduled visits and study procedures.
SMA Participant Exclusion Criteria:
- 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, determined by the study physician
- 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, as established by self-report.
- 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
- Subject has a deconditioned respiratory system, per the discretion of the physician investigator.
- Subjects with renal insufficiency at the discretion of the physician investigator.
- Subjects requiring any form of sedation for MRI will be excluded.
Healthy Control Participant Exclusion Criteria:
- Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator would impact participation in the study.
- Participants who have any serious disease or disorder (e.g. cancer, severe cardiac or respiratory disease, neurological conditions other than stroke, etc.) or cognitive impairments that could affect their ability to participate in this study.
- Female subjects are pregnant or breastfeeding, as established by self-report.
- Subjects requiring sedation for MRI will be excluded.
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 - Upper Limb Protocol
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 2, 3, or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 2, 3, or 4 SMA aged 16 or older that show quantifiable motor deficits of the upper body. The investigators will then implant the subjects with percutaneous, linear spinal leads near the cervical 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 elbow 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.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy 4, SMA
- Being tested
- Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads)
- Keywords
- Spinal Cord Stimulation
- Medical subject headings
- Muscular Atrophy, Spinal, Spinal Muscular Atrophies of Childhood, Spinal muscular atrophy 4
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
Spinal cord stimulation is FDA approved for treatment of pain. We are proposing utilizing the same technology to help restore motor control in people who have declining function due to the progression of SMA. These are 2-4 leads that are FDA-approved.
| Study group | Role | What they receive |
|---|---|---|
| Spinal Cord Stimulation | Experimental | All patients will receive FDA-approved percutaneous spinal cord stimulation leads implanted in the cervical epidural (C4-T1 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) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and Severity of Adverse Events
Success Criteria: no serious adverse events related to the stimulation or intolerable. adverse events reported
Measured: 29 days
Secondary outcomes (14)
Muscle Weakness Muscle Activation
Muscle activation: measure surface electromyogram (EMGs) produced by the subjects during isometric movements of the elbow in the HUMAC Norm and compare to SCS-on with SCS-off and non-affected SCS side performances. Meaningful Change: >20% EMG root-mean-square (RMS) compared to SCS-on.
Measured: 29 days
Motor Function ROM
Range of Motion (ROM): Meaningful Change: Increase of >20% of the elbow joint (if available) during SCS-on against SCS-off and SCS-non affected arm as measured by the HUMAC Norm during single-joint isotonic trials.
Measured: 29 days
Motor Function RULM
The Revised Upper Limb Module (RULM) for SMA is a further refined version of the Hammersmith Functional Motor Scale Extended and includes specific items focused on upper-limb motor control such as raising the arms above the head that are particularly relevant for the Type 2 population of the study, or those with further progressed Type 3 and Type 4. The scale is a performance evaluation with a total score ranging from 0, if none of the activities is achieved, to 37, if all the activities are achieved fully, so a higher score means a better outcome. For our study, we require participants to score at least a 4 to be eligible. Meaningful Change: ≥2 point improvement. Compare outcomes between SCS-on and SCS-off as well as the non-affected arm.
Measured: 29 days
Motor Function Fatigue
Fatigue will be assessed during motor function tests. Patients will be asked to provide a score from 1-10, where a greater number indicates a greater amount of fatigue for each stimulation configuration.
Measured: 29 days
Discomfort/Pain
Patients will be asked to provide a score from 1-10, 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 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 the integrity of the cortico-spinal tract.
Measured: 29 days
Spinal Circuit Excitability
The investigators will measure H-reflexes of the arm 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 arm muscles to calculate the number of firing rates of single spinal motoneuron discharge during isometric maximal voluntary contractions.
Measured: 29 days
Muscle Weakness Torque
Isometric torque: measure the isometric torque produced by the subject at the elbow during flexion and extension as well as grip function during hand held dynamometer as well as during wrist flexion and extension. The investigators will use the measurement of isometric torque to determine the changes in muscle strength for each participant with SCS on and with SCS off. This comparison will also include testing the non-affected arm to give the investigators a baseline for normal movement of the joint being tested. Success Criteria: ≥30% increased torque production over the SCS-off/non-affected baseline as measured during single-joint isometric torque.
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 posted8 Mar 2024
- Study start31 May 2024
- Primary completion19 Oct 2026
- Study completion19 Oct 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies 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
- Not applicable
- 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
- NCT06300996
- Sponsor's protocol ID
- STUDY23110042
- Status last verified
- Oct 2026
- First submitted
- 1 Mar 2024
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: All of the individual participant data collected during the trial, after deidentification, may be shared with other researchers for the purpose of data analysis and collaboration.
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 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 - Upper Limb Protocol
- ClinicalTrials.gov ID
- NCT06300996
- Acronym / sponsor ID
- STUDY23110042
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy 4, SMA
- Keywords
- 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
- 31 May 2024 (actual)
- Primary completion
- 19 Oct 2026 (actual)
- Study completion
- 19 Oct 2026 (actual)
- First posted
- 8 Mar 2024 (actual)
- Results
- Not posted in the source record
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 2, 3, or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 2, 3, or 4 SMA aged 16 or older that show quantifiable motor deficits of the upper body. The investigators will then implant the subjects with percutaneous, linear spinal leads near the cervical 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 elbow 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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 cervical epidural (C4-T1 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 may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
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)Spinal cord stimulation is FDA approved for treatment of pain. We are proposing utilizing the same technology to help restore motor control in people who have declining function due to the progression of SMA. These are 2-4 leads that are FDA-approved. | Device | Spinal Cord Stimulation |
Primary outcome
Number and Severity of Adverse Events
Time frame: 29 days
Success Criteria: no serious adverse events related to the stimulation or intolerable. adverse events reported
14 secondary outcomes
Muscle Weakness Muscle Activation
Time frame: 29 days
Motor Function ROM
Time frame: 29 days
Motor Function RULM
Time frame: 29 days
Motor Function Fatigue
Time frame: 29 days
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
Muscle Weakness Torque
Time frame: 29 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2026.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after deidentification, 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 and Spinal Muscular Atrophy Type 3, 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?
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 23 Oct 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number and Severity of Adverse Events (time frame: 29 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Oct 2026.