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

Not Applicable

Recruiting

• Conditions: Spinal Muscular Atrophy (SMA)

• Registration Number: NCT06977269
• Lead Sponsor: Charitable Foundation Children with Spinal Muscular Atrophy

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-05
• Study First Submitted: 2025-05-09
• Study First Submitted QC: 2025-05-16
• Last Update Submitted: 2025-05-16
• Study First Posted: 2025-05-18
• Last Update Posted: 2025-05-18
• Study Start: 2025-05-19
• Primary Completion: 2025-12
• Study Completion: 2026-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Change from Baseline in the Motor Function Measure Scale	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	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	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

Trial Locations

Locations (1)

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