NCT07578454

Brief Summary

Spinal muscular atrophy is a genetic disorder characterized by progressive muscle weakness, severely impacting patients' motor abilities. Several disease modifying therapies have been developed to treat Spinal muscular atrophy which have led to new disease trajectories . According to standard of care guidelines, exercise programs should be designed and monitored by a physical therapist and should include exercises to improve daily life activities. Exercises should be adapted to each patient and can be prescribed with an optimal frequency in various ways. However, of patients with Spinal muscular atrophy, only 20% reported access to endurance exercises and only 6% to mixed exercises. This incompliance to standard of care guidelines is due to manpower limitation and difficulties in engaging with young and sometimes highly disabled children. Our group has been pioneering in developing the UK at-home individualised rehabilitation program. To address this challenge, the Investigators propose the development of an innovative, virtual targeted rehabilitation platform specifically designed for young patients with Spinal muscular atrophy. This technology aims to provide a patient-centric, at-home rehabilitation solution, enabling parents/caregivers to facilitate daily exercises in a more accessible and enjoyable manner. This technology would constitute the first of its kind in Spinal muscular atrophy field, involving the integration of augmented electromyography signals and soft robotic haptic devices into a gamified virtual reality environment. By increasing the frequency and quality of exercise interventions at home, this technology has the potential to significantly address the critical unmet need for consistent rehabilitation. This technology will also serve as a clinical outcome measure for continuous home-based assessments of weaker and less functional population in place of hospital-based assessments.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for not_applicable

Timeline
9mo left

Started May 2026

Shorter than P25 for not_applicable

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress6%
May 2026Apr 2027

First Submitted

Initial submission to the registry

April 15, 2026

Completed
26 days until next milestone

First Posted

Study publicly available on registry

May 11, 2026

Completed
16 days until next milestone

Study Start

First participant enrolled

May 27, 2026

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2027

Last Updated

June 11, 2026

Status Verified

April 1, 2026

Enrollment Period

10 months

First QC Date

April 15, 2026

Last Update Submit

June 9, 2026

Conditions

Keywords

Spinal Muscular Atrophy

Outcome Measures

Primary Outcomes (2)

  • Cohort 1 (Survey) - To understand current user engagement with exercises and digital games (including time spent and game types) in order to identify and propose suitable activities, games, and tasks for inclusion in the VRehab interface.

    Responses to survey

    At the end of 3 months (+ extension if needed) or completion of 120 surveys in total

  • Cohort 2 (Testing) To evaluate the relevance of a virtual rehabilitation interface for treated patients with SMA

    Number of patients who rate the device as relevant. Each participant will be asked to rate: a. The perceived impact of VRehab SMA device on motor skills using the approved Relevance questionnaire

    through study completion an average of 6 months

Secondary Outcomes (1)

  • Cohort 2 (Testing) - To evaluate the satisfaction/experience of a virtual rehabilitation interface for treated patients with SMA

    through study completion an average of 6 months

Other Outcomes (3)

  • Cohort 2 (Testing) Exploratory objectives - To explore the VRehab device as outcome measure 1

    through study completion an average of 6 months

  • Cohort 2 (Testing) Exploratory objectives - To explore the VRehab device as outcome measure 2

    through study completion an average of 6 months

  • Cohort 2 (Testing) Exploratory objectives -To assess signals of hand strength changes (fatigue).

    through study completion an average of 6 months

Study Arms (2)

Cohort 1(Survey)

NO INTERVENTION

Participants will be asked to complete and online survey with 17 questions (with subparts). The first 4 questions will establish their Spinal muscular atrophy condition and their movement levels. The remaining 13 questions will establish their exercise routines and use of technology in daily life. Followed by their preference for gaming, types of games and TV programs they enjoy. The participants will only be asked once to fill in the survey and this will be carried out at their own homes on their own.

Cohort 2 (Testing)

EXPERIMENTAL

Cohort 2 (Testing) will test out a basic device in a control laboratory environment to determine if further research for long term use and a at home device would be beneficial to patients and not have any adverse effects to their physical health. Participants will be ask to attend a pre-screening video to check eligibility for the study and be consented online at the meeting. Following this meeting 2 visits will be arranged at the University Biomedical engineering lab in Oxford and the participants asked to participate in using the device

Device: VRehab SMA device

Interventions

The VRehab SMA device includes: * A screen to provide visual feedback (like games or animations) * Bluetooth-connected EMG sensors to measure muscle activity in the arms * A ball to measure grip strength * A virtual reality (VR) headset (used only for participants over 10 years of age)

Cohort 2 (Testing)

Eligibility Criteria

Age6 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Person with SMA
  • A person living with genetically confirmed SMA aged from 12-18 years
  • A good understanding of English or someone who can provide a good understanding of English for me to complete the survey
  • A young person living with SMA from 12-15 years of age and who's caregiver/legal guardian gives consent
  • A young person living with SMA from 16-18 years of age who provide their own consent
  • Caregiver/ Legal Guardian
  • A caregiver (legal guardian) of child/young person living with SMA aged from 6-18 years old
  • A good understanding of English or someone who can provide a good understanding of English for me to complete the survey

You may not qualify if:

  • Participant with SMA aged 6-10 years
  • Genetically confirmed SMA
  • A comprehensive understanding of English
  • Treated with any disease-modifying therapy post-symptomatically
  • Number of SMN2 copies available
  • Functional status available
  • Parent(s)/legal guardian(s)/caregiver(s) able to provide written informed consent and child able to provide assent prior to participation in the study
  • Participant with SMA aged 11-15 years
  • Genetically confirmed SMA
  • A comprehensive understanding of English
  • Treated with any disease-modifying therapy post-symptomatically
  • Number of SMN2 copies available
  • Functional status available
  • Parent(s)/legal guardian(s)/caregiver(s) able to provide written informed consent and child able to provide assent prior to participation in the study
  • Participant with SMA aged 16-18 years
  • +7 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Oxford University

Oxford, United Kingdom

RECRUITING

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Central Study Contacts

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
PARALLEL
Model Details: Cohort 1 (Survey) A decentralized and qualitative study that aims to gather data via a survey from participants (minimum N=120) formulated from a combined paediatrics SMA and SMA caregiver population. Cohort 2 (Testing) A prospective and observational study on a total of (N=12) paediatric SMA participants that will partake in testing the VRehab SMA device in a controlled environment.
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 15, 2026

First Posted

May 11, 2026

Study Start

May 27, 2026

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

April 1, 2027

Last Updated

June 11, 2026

Record last verified: 2026-04

Locations