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

Обзор

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.

Вмешательства

• Устройство 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).
• Диагностический тест lumbar puncture
  Cerebrospinal fluid sampling to measure SMN protein and neurofilament concentrations before and after the TMS intervention.

Первичные конечные точки

• Change from Baseline in the Motor Function Measure Scale [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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.]

Критерии участия

Критерии включения

• 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"

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Украина · 1 центр

• P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of — Kharkiv

Идентификаторы

NCT: NCT06977269 · CSMA-INPN-2

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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