Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

VRehab-SMA Phase 1.2

9. juni 2026 opdateret af: 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.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

12

Fase

  • Ikke anvendelig

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Undersøgelse Kontakt Backup

Studiesteder

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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)

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Andet
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Ingen indgriben: 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.
Eksperimentel: 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
Enhed: 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)

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
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.
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

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

Andre resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Cohort 2 (Testing) Exploratory objectives - To explore the VRehab device as outcome measure 1
Tidsramme: 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
Tidsramme: 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).
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

University of Oxford

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

27. maj 2026

Primær færdiggørelse (Anslået)

1. april 2027

Studieafslutning (Anslået)

1. april 2027

Datoer for studieregistrering

Først indsendt

15. april 2026

Først indsendt, der opfyldte QC-kriterier

5. maj 2026

Først opslået (Faktiske)

11. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

11. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

9. juni 2026

Sidst verificeret

1. april 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • Vrehab- PID19470
  • 26/PR/0281 (Anden identifikator: REC)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

UBESLUTET

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Spinal muskelatrofi (SMA)

Kliniske forsøg med VRehab SMA device

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Mali

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner