Clinical trial NCT03127241: User-centred Assistive System for Arm Functions in Neuromuscular Subjects
Official title: USEFUL: User-centred Assistive SystEm for Arm Functions in neUromuscuLar Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Dystrophy, Becker
Muscular Dystrophy, Limb-Girdle Type 2
Keywords: Assistive devices
Randomized controlled trial (RCT)
Upper limbs
Robotic devices
Lead sponsor: Politecnico di Milano
Sponsor class: OTHER
Collaborators: name: IRCCS Eugenio Medea
name: Villa Beretta Rehabilitation Center
First posted: 2017-04-25
Enrollment: 38
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: SUPPORTIVE_CARE

Brief summary: Restore a lost function is a special experience for people affected by neuromuscular evolutive diseases. "From the patient's point of view improvement is measured by regaining lost abilities,-by being able to do something -anything-today I couldn't do yesterday ". Upper limb pain, stiffness and activity limitations have a crucial role in reducing patients' autonomy and worsening quality of life.

Real users' needs have been identified thought several workshops, and even if the commercial products might assure a benefit to some users and meet most of their requirements, so far a validation of the use of such devices by people with neuromuscular disease is missing.

We aim at field-testing the improvement in arm functions provided by the use of some commercial devices and assessing their impact to users' quality of life and independence. This step is essential to assure a widespread accessibility to these devices for most of the potential users, possibly providing health providers with direction and guidance towards Health Technology Assessment.

Clinical Trial design - The study proposes on-field validation of JAECO WREX, passive antigravity exoskeleton; and Armon Ayura, motorized arm exoskeleton for gravity compensation in a randomized controlled trial with crossover design. The clinical study will is multi-centric, involving both MEDEA and VALDUCE, and received the Ethical Committee approval.


Eligibility criteria: Inclusion criteria

* signed informed consent (we will expose the family the aims of our study and carefully describe the need of compliance with them)
* diagnosis of muscular dystrophy (genetic, histological and biochemical if necessary),specifically with Duchenne, Becker and Limb Girdle type 2 muscular dystrophy. No muscle biopsy will be performed, except in case there is a diagnostic need.
* being wheelchair bound
* having a scoring at the MRC scale for upper limb at the deltoid, biceps, triceps brachii muscles ranging from 2 to 4.

Exclusion criteria

* presence of additional diseases (for example: family history of mental retardation, epilepsy, cerebral palsy)
* behavioral and psychiatric disturbances (emotional problems, depression)
* absence of compliance to the study of the family and the caregivers

Primary outcomes:
measure: Performance of the Upper Limb (PUL) scale
timeFrame: T0 (baseline); T1 (after 3-days short training with device 1); T2 (after 2-weeks home use of device 1 - primary assessment point); T3 (after 3-days short training with device 2); T4 (after 2-weeks home use of device 2 - secondary assessment point)
Secondary outcomes:
measure: Motor Function Measures scale (MFM)
timeFrame: T0 (baseline); T1 (after 3-days short training with device 1); T2 (after 2-weeks home use of device 1 - primary assessment point); T3 (after 3-days short training with device 2); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: Brooke scale
timeFrame: T0 (baseline); T1 (after 3-days short training with device 1); T2 (after 2-weeks home use of device 1 - primary assessment point); T3 (after 3-days short training with device 2); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: ABILHAND
timeFrame: T0 (baseline); T1 (after 3-days short training with device 1); T2 (after 2-weeks home use of device 1 - primary assessment point); T3 (after 3-days short training with device 2); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: PedsQL
timeFrame: T0 (baseline); T2 (after 2-weeks home use of device 1 - primary assessment point); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: PROMIS FATIGUE Short Form
timeFrame: T0 (baseline); T2 (after 2-weeks home use of device 1 - primary assessment point); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: Personal Adjustment and Role Skills Scale (PARS) III
timeFrame: T0 (baseline); T2 (after 2-weeks home use of device 1 - primary assessment point); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: Technology Acceptance Model (TAM)
timeFrame: T2 (after 2-weeks home use of device 1 - primary assessment point); T3 (after 3-days short training with device 2); T4 (after 2-weeks home use of device 2 - secondary assessment point)
measure: System Usability Scale (SUS)
timeFrame: T2 (after 2-weeks home use of device 1 - primary assessment point); T3 (after 3-days short training with device 2); T4 (after 2-weeks home use of device 2 - secondary assessment point)
Other outcomes:
Locations:
city: Bosisio Parini
country: Italy
facility: IRCCS E. Medea - La Nostra Famiglia
state: LC
city: Costa Masnaga
country: Italy
facility: Villa Beretta
state: LC
Study officials:
affiliation: Politecnico di Milano
name: Alessandra Pedrocchi, Prof
role: PRINCIPAL_INVESTIGATOR
References:
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