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

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

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

Study Overview

• Status: Recruiting
• Conditions: SMA
• Intervention / Treatment: Procedure: Robotically Assisted Locomotion; Procedure: Robotically Assisted Verticalization

• Study Type: Interventional
• Enrollment (Estimated): 200
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Marek Jóźwiak; Phone Number: +48 696052475; Email: sma@orsk.pl
• Study Contact Backup: Name: Radosław Rutkowski; Phone Number: 696052475; Email: sma@orsk.pl

Study Locations

• Poland
  • Poznań, Poland, 61-545
    • Recruiting
    • Wiktor Dega University Orthopedic and Rehabilitation Hospital
    • Contact: Marek Jóźwiak; Phone Number: +48 696052475; Email: sma@orsk.pl
    • Contact: Radosław Rutkowski; Phone Number: 696052475; Email: sma@orsk.pl

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Robotically Assisted Locomotion	Procedure: Robotically 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
Active Comparator: Robotically Assisted Verticalization	Procedure: Robotically Assisted Verticalization; Motor control training; Verticalization training with Standard Frame Devices; Locomotion and motor control training with elements of balance and coordination; Cognitive therapy

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Wiktor Dega University Orthopedic and Rehabilitation Hospital
• Investigators: Principal Investigator: Marek Jóźwiak, Wiktor Dega University Orthopedic and Rehabilitation Hospital

Study record dates

Study Major Dates

• Study Start (Actual): December 21, 2023
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): July 1, 2027

Study Registration Dates

• First Submitted: January 26, 2024
• First Submitted That Met QC Criteria: March 13, 2024
• First Posted (Actual): March 21, 2024

Study Record Updates

• Last Update Posted (Actual): March 21, 2024
• Last Update Submitted That Met QC Criteria: March 13, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: SMA; Robotically-assisted therapy; Robotically Assisted Locomotion; Robotically Assisted Verticalization
• Other Study ID Numbers: 2023/ABM/01/00004/P/02

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No