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

Not Applicable

Recruiting

• Conditions: SMA

• Registration Number: NCT06322654
• Lead Sponsor: Wiktor Dega University Orthopedic and Rehabilitation Hospital

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

Detailed Description

Not available

Study Timeline

• Study Start: 2023-12-21
• Study First Submitted: 2024-01-26
• Status Verified: 2024-03
• Study First Submitted QC: 2024-03-13
• Last Update Submitted: 2024-03-13
• Study First Posted: 2024-03-21
• Last Update Posted: 2024-03-21
• Primary Completion: 2027-03-31
• Study Completion: 2027-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 200

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
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
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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Wiktor Dega University Orthopedic and Rehabilitation Hospital; 🇵🇱 Poznań, Poland