Congenital Myopathies, Dystrophinopathy, Muscular Dystrophies
Conditions
Keywords
Sports for Persons with Disabilities, Rehabilitation, Neuromuscular Diseases, Dual-Task Exercises, Virtual Reality, Children with Disabilities
Brief summary
The goal of this clinical trial is to assess the effects of a dual-task, multimodal training program on slowing functional motor decline in children and adolescents with neuromuscular disorders. The main questions it aims to answer are: Does Virtual Park slow functional motor decline in children and adolescents with neuromuscular diseases compared with standard therapy? Does Virtual Park contribute to increased engagement and improved neuropsychological function? If there is a comparison group, Researchers will compare dual-task cycling supported by the Virtual Park application to standard therapy to see whether it improves motor and neuropsychological functions and engagement. Participants will undergo two phases: * Phase 1: 3 months of standard therapy; * Phase 2: 3 months of experimental conditions in which patients undergo a rehabilitative intervention with Virtual Park, 3 times a week.
Interventions
DEVICEDual-task cycling
VirtualPark is a software application connecting to a medical certified cycle/arm ergometer (i.e., TheraTrainer TIGO). The application allows for dual task training. In particular, while cycling, participants can perform one of four different available exercises. Each exercise is focused on a specific cognitive domain: Task 1: visual attention, inhibition and cognitive flexibility; Task 2: working memory; Task 3: inhibition; Task 4: navigation.
OTHERStandard therapy
Participants undergo their standard plan: outpatient treatment, if planned, or no treatment.
Sponsors
Istituto di Sistemi e Tecnologie Industriali Intelligenti per il Manifatturiero Avanzato
IRCCS National Neurological Institute "C. Mondino" Foundation
IRCCS Eugenio Medea
Fondazione Don Carlo Gnocchi ETS
IRCCS Fondazione Stella Maris
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Intervention model description
Participants first undergo 3 months of standard therapy, followed by 3 months of intervention with Virtual Park. They act as controls for themselves.
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* availability of a definitive clinical or molecular genetic diagnosis of a neuromuscular disease; * age between 6 years and 17 years and 11 months (inclusive); * height compatible with the use of the Thera-Trainer (minimum 120 cm); * performance Upper Limb Module 2.0 assessment: Entry level \>= 2
Exclusion criteria
* other concomitant genetic diseases; * presence of visual impairments that prevent access to the experimental virtual reality protocol; * concomitant compromised cardio-respiratory function that contraindicates cycle ergometer training; * severe osteoporosis.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Motor Function Measure-32 (MFM-32) - Total score | Baseline (pre-treatment), 3 months (post-standard therapy, T1), 6 months (post-VirtualPark intervention, T2) | The Motor Function Measure-32 is a motor skills assessment scale validated for patients affected by neuromuscular pathology from 6 years of age. The instrument consists of 32 items (scored from 0 to 3) divided into 3 dimensions: D1- standing position and transfers (13 items), D2- axial and proximal motor skills (12 items) and D3- distal motor skills (7 items). The results are expressed as a percentage in relation to the maximum score. The result of each dimension corresponds to the sum of the scores obtained by the subject in the items of this dimension divided by the maximum score of the same dimension and multiplied by 100. For the MFM-32 the total score corresponds to the sum of all the scores attributed to the subject (all dimensions together) divided by 96 and multiplied by 100. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| North Star Ambulatory Assessment (NSSA) | Baseline (pre-treatment), 3 months (post-standard therapy, T1), 6 months (post-VirtualPark intervention, T2) | \[FOR AMBULATORY PARTICIPANTS ONLY\] The North Star Ambulatory Assessment (NSSA) scale was developed specifically for ambulatory children with dystrophinopathy starting at age 5 to obtain information on their motor and functional abilities. The instrument consists of 17 items scored from 0 to 2. The final score is the sum of the scores obtained for all items. Two timed tests are also recorded: the time to stand up from the ground and the time required to walk/run 10 meters. |
| Timed functional tests (TFTs) | Baseline (pre-treatment), 3 months (post-standard therapy, T1), 6 months (post-VirtualPark intervention, T2) | \[FOR AMBULATORY PARTICIPANTS ONLY\] Timed functional tests (TFTs) are administered in a standardized manner to subjects with Duchenne muscular dystrophy to measure the time taken to perform the following four activities: moving from a supine to a standing position, walking/running for 10 meters, and ascending and descending a standardized 4-step staircase. The scoresheet records, in addition to the time taken to complete the task, a qualitative grading score from 1 to 6. |
| 6-Minute Walking Test (6MWT) | Baseline (pre-treatment), 3 months (post-standard therapy, T1), 6 months (post-VirtualPark intervention, T2) | \[FOR AMBULATORY PARTICIPANTS ONLY\] The 6-Minute Walking Test (6MWT) was developed in the cardio-respiratory field but is now used across various fields of motor rehabilitation where it is necessary to quantify the patient's walking endurance. The test measures the distance a patient can cover, walking on a flat surface along a standardized path, in a period of 6 minutes \[25\]. In addition to the total distance covered, the distances covered for each minute are also collected and any falls/pauses are recorded. |
| Performance Upper Limb Module 2.0 scale (PUL version 2.0) | Baseline (pre-treatment), 3 months (post-standard therapy, T1), 6 months (post-VirtualPark intervention, T2) | \[FOR NON-AMBULATORY PARTICIPANTS ONLY\] The Performance Upper Limb Module 2.0 scale (PUL version 2.0) was developed to evaluate the motor performance of the upper limbs of subjects with dystrophinopathies from 5 years of age. This latest version of the instrument consists of an entry item useful for defining the patient's functional level and scored separately (from 0 to 6) and of 22 other items (scored from 0 to 2). The final score is made up of 2 values: the entry level score and the total score given by the sum of the scores obtained in the individual items \[26\]. |
| Assisted-6 Minute Cycling Test (A6MWT) | Baseline (pre-treatment), 3 months (post-standard therapy, T1), 6 months (post-VirtualPark intervention, T2) | Performance change in the Assisted 6-minute cycling test (A6MCT). The A6MCT is a submaximal endurance test performed by pedaling with a cycle ergometer in passive mode for both upper and lower limbs, specifically created for subjects affected by Duchenne muscular dystrophy, both ambulatory and non-ambulatory. The final score records the number of complete revolutions performed in 6 minutes; the number of revolutions per minute and any breaks requested by the subject are also recorded \[27\]. |
Countries
Italy
Contacts
CONTACTSara Arlati, Bioengineer
Outcome results
None listed