VRehab-SMA Phase 1.2

Official title

Virtual Targeted Rehabilitation for Patients With Spinal Muscular Atrophy: Phase 1.2: Proof-of-concept

Overview

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.

Detailed description

Spinal muscular atrophy is a genetic disorder where muscles progressively weaken, severely impacting patients' motor abilities eg sitting or walking etc.

Several disease modifying therapies have been developed to treat Spinal muscular atrophy which have led to new disease pathways. For example, previously patients who could not sit can now sit but still not walk. 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 in a recent survey, of patients with Spinal muscular atrophy, only 20% report access to endurance exercises and only 6% report mixed exercises. This is due to manpower limitation and difficulties in engaging with young and sometimes highly disabled children.

To address this, 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 caregivers to facilitate daily exercises in a more accessible and enjoyable manner. This technology would involve the integration of augmented electromyography signals and soft robotic haptic devices into a game controlled by exercise/movement for children. By increasing the frequency and quality of exercise interventions at home, this technology has the potential to significantly address the critically unmet need for consistent rehabilitation and decrease the burden of hospital-based assessments.

This is a proof of concept study to assess patients with Spinal muscular atrophy desire to use the equipment and the potential benefit this can bring to patients by surveying Spinal muscular atrophy patients about technology and exercise and testing the current equipment in a lab setting.

Interventions

• Device VRehab SMA device
  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)

Primary outcome measures

• 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. [Time frame: 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 [Time frame: through study completion an average of 6 months]

Secondary outcome measures (1)

• Cohort 2 (Testing) - To evaluate the satisfaction/experience of a virtual rehabilitation interface for treated patients with SMA [Time frame: through study completion an average of 6 months]

Eligibility criteria

Cohort 1 Inclusion Criteria:

• 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

Exclusion Criteria: No exclusion criteria for Cohort 1, other than meeting the inclusion criteria

Cohort 2 Inclusion Criteria:

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
• Age: Participants between 6-10 years at Visit 1 (inclusion)
• 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
• Age: Participants between 11-15 years at Visit 1 (inclusion)
• 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

• Genetically confirmed SMA
• A comprehensive understanding of English
• Treated with any disease-modifying therapy post-symptomatically
• Number of SMN2 copies available
• Functional status available
• Age: Participants between 16-18 years at Visit 1 (inclusion)
• Able to provide written informed consent

Exclusion criteria

Any acute or chronic condition which, according to the investigator, significantly interferes with the use of the device (example: Upper limbs injuries interfering with technology, skin conditions preventing the use of electrodes, etc)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Non-randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Other

Study locations

United Kingdom · 1 center

• Oxford University — Oxford

Identifiers

NCT: NCT07578454 · Vrehab- PID19470 · 26/PR/0281

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗