Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy (STIM-SMA)

Charitable Foundation Children with Spinal Muscular Atrophy

Status: Enrolling

Conditions: Spinal Muscular Atrophy (SMA)

Treatments: Diagnostic Test: lumbar puncture; Device: High-frequency repetitive transcranial magnetic stimulation

Study type: Interventional

Funder types: Other

Identifiers

NCT06977269

CSMA-INPN-2

Details and patient eligibility

About

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.

Enrollment: 20 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

TMS arm

Experimental group

Description:

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.

Treatment:

Device: High-frequency repetitive transcranial magnetic stimulation

Diagnostic Test: lumbar puncture