Clinical trial NCT06977269: Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
Official title: Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Spinal Muscular Atrophy (SMA)
Keywords: spinal muscular atrophy
Survival Motor Neuron protein (SMN)
Neurofilament (NF)
transcranial magnetic stimulation (TMS)
Lead sponsor: Charitable Foundation Children with Spinal Muscular Atrophy
Sponsor class: OTHER
Collaborators: name: P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
First posted: 2025-05-18
Enrollment: 20
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change from Baseline in the Motor Function Measure Scale
timeFrame: 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.
measure: Change from Baseline of the Revised Upper Limb Module
timeFrame: 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.
measure: Change from Baseline of the Hammersmith Functional Motor Scale - Expanded
timeFrame: 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.
measure: Change from Baseline in the 6-Minute Walk Test
timeFrame: 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.
Secondary outcomes:
Other outcomes:
Locations:
city: Kharkiv
country: Ukraine
facility: P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
Study officials:
affiliation: P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
name: Olena V Pisotska, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: P.V. Voloshyn Institute of Neurology, Psychiatry and Narcology of the National Academy of Medical Sciences of Ukraine
name: Andriy V Shatillo, MD, PhD
role: STUDY_CHAIR
References: