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

NCT06322654 | Recruiting DMC

• 1 other identifier: 2023/ABM/01/00004/P/02
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 1

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

Conditions

SMA

Keywords

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

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (3)

• 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

Study Arms (2)

Robotically Assisted Locomotion

EXPERIMENTAL

Procedure: Robotically Assisted Locomotion

Robotically Assisted Verticalization

ACTIVE COMPARATOR

Procedure: Robotically Assisted Verticalization

Interventions

Robotically Assisted Locomotion PROCEDURE

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

Robotically Assisted Locomotion

Robotically Assisted Verticalization PROCEDURE

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

Robotically Assisted Verticalization

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Wiktor Dega University Orthopedic and Rehabilitation Hospital: lead

Study Sites (1)

Wiktor Dega University Orthopedic and Rehabilitation Hospital

Poznan, 61-545, Poland

RECRUITING

Study Officials

• Marek Jóźwiak

  Wiktor Dega University Orthopedic and Rehabilitation Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Marek Jóźwiak

CONTACT

696052475; sma@orsk.pl

Radosław Rutkowski

CONTACT

696052475; sma@orsk.pl

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Masking Details: 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.
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: January 26, 2024
• First Posted: March 21, 2024
• Study Start: December 21, 2023
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: March 21, 2024

Record last verified: 2024-03

Locations

PL (1)