Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy

NCT ID: NCT06977269

Last Updated: 2025-06-18

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-19
• Study Completion Date: 2026-01-31

Brief 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 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)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

TMS arm

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.

Group Type: EXPERIMENTAL

High-frequency repetitive transcranial magnetic stimulation

Intervention Type: DEVICE

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 puncture

Intervention Type: DIAGNOSTIC_TEST

Cerebrospinal fluid sampling to measure SMN protein and neurofilament concentrations before and after the TMS intervention.

Interventions

High-frequency repetitive transcranial magnetic stimulation

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).

Intervention Type: DEVICE

lumbar puncture

Cerebrospinal fluid sampling to measure SMN protein and neurofilament concentrations before and after the TMS intervention.

Intervention Type: DIAGNOSTIC_TEST

Other Intervention Names

HF-rTMS

Eligibility Criteria

Inclusion Criteria

• 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"

Exclusion Criteria

• 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.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine

UNKNOWN

Sponsor Role: collaborator

Charitable Foundation Children with Spinal Muscular Atrophy

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Olena V Pisotska, MD, PhD

Role: 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

Role: STUDY_CHAIR

P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine

Locations

P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine

Kharkiv, Kharkivs’ka Oblast’, Ukraine

Site Status: RECRUITING

Countries

Ukraine

Central Contacts

Vitaliy M Matyushenko

Role: CONTACT

csma.ua@gmail.com

380503640673

Andriy V Shatillo, MD, PhD

Role: CONTACT

shatil@ukr.net

380978111884

Facility Contacts

Olena Pisotska Medical Director, MD, PhD

Role: primary

inpn@ukr.net

380577384194

Other Identifiers

CSMA-INPN-2

Identifier Type: -

Identifier Source: org_study_id