A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients

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

Status

Recruiting

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06322654

Acronym

SMArt

Enrollment

200

Registered

2024-03-21

Start date

2023-12-21

Completion date

2027-07-31

Last updated

2024-03-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

SMA

Keywords

SMA, Robotically-assisted therapy, Robotically Assisted Locomotion, Robotically Assisted Verticalization

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

Interventions

PROCEDURERobotically Assisted Locomotion

* Motor control training * Locomotion and motor control training with elements of balance and coordination * Verticalization training: standing frame device and vibration platform * Cognitive therapy

PROCEDURERobotically Assisted Verticalization

* Motor control training * Verticalization training with Standard Frame Devices * Locomotion and motor control training with elements of balance and coordination * Cognitive therapy

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

SUPPORTIVE_CARE

Masking

SINGLE (Investigator)

Masking description

Patients and their parents will not be blinded by the nature of the therapeutic procedure used after random allocation to the treatment group. Study personnel will be divided into Blinded Team and Unblinded Team to ensure objectivity of the assessment of safety and effectiveness of both strategies.

Intervention model description

There are two research groups with 100 patients in each group. 1. Experimental: Robotically Assisted Locomotion 2. Comparison: Robotically Assisted Verticalization

Eligibility

Sex/Gender

ALL

Age

No minimum to 21 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale	Through study completion, max. for 2.5 year every 6 months
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.	Through study completion, max. for 2.5 year every 6 months
Functional assessment of patients using GMFM (Gross Motor Function Measure) scale	Through study completion, max. for 2.5 year every 6 months
X-ray or ultrasound scan imaging assessment of the spine, hip joints and bone mineral density	Once per 1 year
Assessment of the quality of life of patients and their caregivers using Pediatric Quality of Life Inventory ver. 4.0	Through study completion, max. for 2.5 year every 6 months

Secondary

Measure	Time frame
Hospitalization during or between rehabilitations that take place each 6 months	Through study completion, max. for 2.5 year
Occurence of discomfort during therapy requiring abrupt interruption or significant modification	Through study completion, max. for 2.5 year every 6 months
Number of fractures	Through study completion, max. for 2.5 year

Countries

Poland

Contacts

Primary ContactMarek Jóźwiak

sma@orsk.pl696052475

Backup ContactRadosław Rutkowski