Spinal Muscular Atrophy (SMA)STIM-SMANCT06977269
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
A study testing High-frequency repetitive transcranial magnetic stimulation and lumbar puncture in people with Spinal Muscular Atrophy (SMA), run by Charitable Foundation Children with Spinal Muscular Atrophy at 1 site in Ukraine.
Trial summary
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration. Although the relevance of that rule to exercises in neuromuscular patients and for SMA in particular is not definitely proven, clinical observations seem to support this… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Spinal Muscular Atrophy (SMA)
- What is being tested?
- High-frequency repetitive transcranial magnetic stimulation and lumbar puncture
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUkraine
- Study dates
- 19 May 2025to Jan 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by DNA test.
- Informed consent.
- 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"
Who cannot join
- Subject has severe joint contractures that would affect ability to perform study measures, determined by the study physician.
- Subject has a deconditioned respiratory system, per the discretion of the physician investigator.
- Subject has behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Ukraine1 site, 1 recruiting
- Recruiting
P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
Kharkiv, Kharkiv Oblast, 61068
Olena Pisotska Medical Director, MD, PhD, 380577384194, inpn@ukr.net
About this study
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration. Although the relevance of that rule to exercises in neuromuscular patients and for SMA in particular is not definitely proven, clinical observations seem to support this assumption. Also there are several experimental studies which provide additional support for utility of exercise for SMA.
However, making regular exercises may be very challenging with SMA not only due to physical limitations, but due to psychological either.
While being considered as safe and well tolerated intervention, TMS is able to mimic effects of real physical exercises, at least at the level of low motoneuron, it also provides several advantages. For example, possibility to exercise non-collaborative infants, minimization of psychological motivation impact in adults and/or ability to involve very weak muscle groups.
- Conditions
- Spinal Muscular Atrophy (SMA)
- Being tested
- High-frequency repetitive transcranial magnetic stimulation, lumbar puncture
- Keywords
- spinal muscular atrophy, Survival Motor Neuron protein (SMN), Neurofilament (NF), transcranial magnetic stimulation (TMS)
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
High-frequency repetitive transcranial magnetic stimulationDevice
Also called HF-rTMS
High-frequency repetitive transcranial magnetic stimulation targeting the primary motor cortex (M1) of the limbs, delivered at a frequency above 5 Hz and an intensity of 90-100% of the resting motor threshold, across 10 sessions with up to 2400 stimuli per session, is a standard intervention used in various neurological disorders. However, its effects have not been studied in patients with spinal muscular atrophy (SMA).
lumbar punctureDiagnostic test
Cerebrospinal fluid sampling to measure SMN protein and neurofilament concentrations before and after the TMS intervention.
| Study group | Role | What they receive |
|---|---|---|
| TMS arm | Experimental | Stimulation (HF-rTMS) of the primary motor cortex, M1 area of the limbs, at a frequency above 5 Hz and an intensity of 90-100% of the resting motor threshold (RMT), over 10 sessions, up to 2400 stimuli per session. Receives: Device: High-frequency repetitive transcranial magnetic stimulation; Diagnostic Test: lumbar puncture |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in the Motor Function Measure Scale
Motor Function Measure (MFM) scale measurement before and after TMS sessions
Measured: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Change from Baseline of the Revised Upper Limb Module
Revised Upper Limb Module (RULM) scale measurement before and after TMS sessions
Measured: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Change from Baseline of the Hammersmith Functional Motor Scale - Expanded
Hammersmith Functional Motor Scale - Expanded (HFMSE) measurement before and after TMS sessions
Measured: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Change from Baseline in the 6-Minute Walk Test
6-Minute Walk Test (6MWT) measurement before and after TMS sessions
Measured: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2025
- Study start19 May 2025
- Primary completionDec 2025 (estimated)
- Study completionJan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy (SMA)Studies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
- Sponsor type
- University, hospital or other
- Collaborators
- P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
- Responsible party
- Sponsor
- Investigators
- Olena V Pisotska, MD, PhD, Principal investigator, P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of UkraineAndriy V Shatillo, MD, PhD, Study chair, P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06977269
- Sponsor's protocol ID
- CSMA-INPN-2
- Acronym
- STIM-SMA
- Status last verified
- Jun 2025
- First submitted
- 9 May 2025
- Last updated
- 18 Jun 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: only IPD used in the results publication
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT06977269
- Acronym / sponsor ID
- STIM-SMA / CSMA-INPN-2
- Conditions
- Spinal Muscular Atrophy (SMA)
- Keywords
- spinal muscular atrophy, Survival Motor Neuron protein (SMN), Neurofilament (NF), transcranial magnetic stimulation (TMS)
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Charitable Foundation Children with Spinal Muscular AtrophyUniversity, hospital or other
- Collaborators
- P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
- Study start
- 19 May 2025 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Jan 2026 (estimated)
- First posted
- 18 May 2025 (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.
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration. Although the relevance of that rule to exercises in neuromuscular patients and for SMA in particular is not definitely proven, clinical observations seem to support this assumption. Also there are several experimental studies which provide additional support for utility of exercise for SMA.
However, making regular exercises may be very challenging with SMA not only due to physical limitations, but due to psychological either.
While being considered as safe and well tolerated intervention, TMS is able to mimic effects of real physical exercises, at least at the level of low motoneuron, it also provides several advantages. For example, possibility to exercise non-collaborative infants, minimization of psychological motivation impact in adults and/or ability to involve very weak muscle groups.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TMS arm | Experimental | Device: High-frequency repetitive transcranial magnetic stimulation, Diagnostic Test: lumbar punctureStimulation (HF-rTMS) of the primary motor cortex, M1 area of the limbs, at a frequency above 5 Hz and an intensity of 90-100% of the resting motor threshold (RMT), over 10 sessions, up to 2400 stimuli per session. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by DNA test.
- Informed consent.
- 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"
Key exclusion criteria (3 listed)
- Subject has severe joint contractures that would affect ability to perform study measures, determined by the study physician.
- Subject has a deconditioned respiratory system, per the discretion of the physician investigator.
- Subject has behavioral or cognitive problems that preclude participation in the study, 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 |
|---|---|---|
| High-frequency repetitive transcranial magnetic stimulationAlso: HF-rTMSHigh-frequency repetitive transcranial magnetic stimulation targeting the primary motor cortex (M1) of the limbs, delivered at a frequency above 5 Hz and an intensity of 90-100% of the resting motor threshold, across 10 sessions with up to 2400 stimuli per session, is a standard intervention used in various neurological disorders. However, its effects have not been studied in patients with spinal muscular atrophy (SMA). | Device | TMS arm |
| lumbar punctureCerebrospinal fluid sampling to measure SMN protein and neurofilament concentrations before and after the TMS intervention. | Diagnostic test | TMS arm |
Primary outcome
Change from Baseline in the Motor Function Measure Scale
Time frame: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Motor Function Measure (MFM) scale measurement before and after TMS sessions
Change from Baseline of the Revised Upper Limb Module
Time frame: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Revised Upper Limb Module (RULM) scale measurement before and after TMS sessions
Change from Baseline of the Hammersmith Functional Motor Scale - Expanded
Time frame: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Hammersmith Functional Motor Scale - Expanded (HFMSE) measurement before and after TMS sessions
Change from Baseline in the 6-Minute Walk Test
Time frame: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
6-Minute Walk Test (6MWT) measurement before and after TMS sessions
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2025.
- Individual participant dataSharing planned: Yes. only IPD used in the results publication
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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.
Olena V Pisotska, MD, PhD principal investigator
P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
Andriy V Shatillo, MD, PhD study chair
P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing High-frequency repetitive transcranial magnetic stimulation and lumbar puncture in people with Spinal Muscular Atrophy (SMA), run by Charitable Foundation Children with Spinal Muscular Atrophy at 1 site in Ukraine.
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 3 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 18 Jun 2025. 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 Ukraine. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Vitaliy M Matyushenko (380503640673, csma.ua@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in the Motor Function Measure Scale (time frame: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.). The study lists 4 primary outcomes in all. 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 18 Jun 2025.