Clinical trial NCT06322654: A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients
Official title: A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA Patients: Single-center, Randomized, Single-blinded, Comparative Study of a Robotically Active Verticalization Model Versus Robotically Assisted Locomotion Model
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: SMA
Keywords: SMA
Robotically-assisted therapy
Robotically Assisted Locomotion
Robotically Assisted Verticalization
Lead sponsor: Wiktor Dega University Orthopedic and Rehabilitation Hospital
Sponsor class: OTHER
First posted: 2024-03-21
Enrollment: 200
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: There are two research groups with 100 patients in each group.

1. Experimental: Robotically Assisted Locomotion
2. Comparison: Robotically Assisted Verticalization
Primary purpose: SUPPORTIVE_CARE

Brief summary: A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization versus Robotically Assisted Locomotion.

The objective of research:

The main goal of the project is to determine the optimal robotically assisted rehabilitation model for people with SMA depending on age and baseline functional status. The study consists of a head-to-head comparison of two rehabilitation models.

1. Research period: 4 years
2. Patients age: 0-21 y.o.
3. Group size: 200 patients (100 patients in each group)
4. Assignment of patients to study groups in a randomised manner


Eligibility criteria: Inclusion Criteria:

* Signing of informed consent to participate in the experiment by the participant or the participant's parent/legal guardian
* Diagnosis of SMA (spinal muscular atrophy 1-4) confirmed by genetic testing, symptomatic or pre-symptomatic
* Age between 0 and 21 years of age (will be determined on the day of starting participation in the project based on the date of birth)
* Treatment under a drug program for spinal muscular atrophy, provided that the patient has a diagnosis of spinal muscular atrophy at the date of eligibility.

Exclusion Criteria:

* Cardio-respiratory disorders requiring invasive ventilation
* Advanced osteoporosis with multiple fractures prior to treatment
* Functional deterioration during the rehabilitation process in the range of scales appropriately selected for the SMA type: Prechtl, HINE, CHOP-INTEND, HFMS, RULM
* Lack of cooperation with the therapist
* Other functional indications preventing exercise

Primary outcomes:
measure: Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale
timeFrame: Through study completion, max. for 2.5 year every 6 months
measure: Clinical assessment of patients using Balance test minimum 1 min., Self-walk test of at least 2 meters, 6-minute gait test, Up&Go test, 10 meters gait test, hip range of motion and muscle strength evaluation.
timeFrame: Through study completion, max. for 2.5 year every 6 months
measure: Functional assessment of patients using GMFM (Gross Motor Function Measure) scale
timeFrame: Through study completion, max. for 2.5 year every 6 months
measure: X-ray or ultrasound scan imaging assessment of the spine, hip joints and bone mineral density
timeFrame: Once per 1 year
measure: Assessment of the quality of life of patients and their caregivers using Pediatric Quality of Life Inventory ver. 4.0
timeFrame: Through study completion, max. for 2.5 year every 6 months
Secondary outcomes:
measure: Hospitalization during or between rehabilitations that take place each 6 months
timeFrame: Through study completion, max. for 2.5 year
measure: Occurence of discomfort during therapy requiring abrupt interruption or significant modification
timeFrame: Through study completion, max. for 2.5 year every 6 months
measure: Number of fractures
timeFrame: Through study completion, max. for 2.5 year
Other outcomes:
Locations:
city: Poznań
country: Poland
facility: Wiktor Dega University Orthopedic and Rehabilitation Hospital
Study officials:
affiliation: Wiktor Dega University Orthopedic and Rehabilitation Hospital
name: Marek Jóźwiak
role: PRINCIPAL_INVESTIGATOR
References: