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VRehab-SMA Phase 1.2

14 september 2026 bijgewerkt door: University of Oxford

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

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.

Studie Overzicht

Toestand

Actief, niet wervend

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Geschat)

12

Fase

  • Niet toepasbaar

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Kind
  • Volwassen

Accepteert gezonde vrijwilligers

Nee

Beschrijving

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)

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Ander
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Geen tussenkomst: Cohort 1(Survey)
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.
Experimenteel: Cohort 2 (Testing)
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
Apparaat: 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)

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
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.
Tijdsspanne: At the end of 3 months (+ extension if needed) or completion of 120 surveys in total
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
Tijdsspanne: through study completion an average of 6 months

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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Cohort 2 (Testing) - To evaluate the satisfaction/experience of a virtual rehabilitation interface for treated patients with SMA
Tijdsspanne: through study completion an average of 6 months

Satisfaction/experience questionnaire. Each participant will be asked to rate:

  1. Feedback on device
  2. Usability
  3. Areas of improvement
through study completion an average of 6 months

Andere uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Cohort 2 (Testing) Exploratory objectives - To explore the VRehab device as outcome measure 1
Tijdsspanne: through study completion an average of 6 months

1. Outcome measures used as anchors:

a, Revised upper limb module (RULM): Total score and range of motion as measured by motion capture cameras at V1 and compared with range of motion captured during the use of VRehab SMA device
through study completion an average of 6 months
Cohort 2 (Testing) Exploratory objectives - To explore the VRehab device as outcome measure 2
Tijdsspanne: through study completion an average of 6 months
Myogrip: measured strength value and compare with strength captured during the use of VRehab SMA device
through study completion an average of 6 months
Cohort 2 (Testing) Exploratory objectives -To assess signals of hand strength changes (fatigue).
Tijdsspanne: through study completion an average of 6 months
A Comparison of the pattern and range of maximum grip strength (maximum voluntary contraction (MVC) between the first few visit trials and last few visit trials
through study completion an average of 6 months

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

University of Oxford

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

27 mei 2026

Primaire voltooiing (Geschat)

1 april 2027

Studie voltooiing (Geschat)

1 april 2027

Studieregistratiedata

Eerst ingediend

15 april 2026

Eerst ingediend dat voldeed aan de QC-criteria

5 mei 2026

Eerst geplaatst (Werkelijk)

11 mei 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

15 september 2026

Laatste update ingediend die voldeed aan QC-criteria

14 september 2026

Laatst geverifieerd

1 april 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • Vrehab- PID19470
  • 26/PR/0281 (Andere identificatie: REC)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

ONBESLIST

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

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