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

SMA Clinical Trial

— SMArt  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06322654
• Other study ID #: 2023/ABM/01/00004/P/02
• Status: Recruiting
• Phase: N/A
• Start date: December 21, 2023
• Est. completion date: July 2027

Study information

• Verified date: March 2024
• Source: Wiktor Dega University Orthopedic and Rehabilitation Hospital
• Contact: Marek Józwiak
• Phone: 696052475
• Email: sma[@]orsk.pl
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: July 2027
• Est. primary completion date: March 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• SMA

Intervention

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
• 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

Locations

Country	Name	City	State
Poland	Wiktor Dega University Orthopedic and Rehabilitation Hospital	Poznan

Sponsors (1)

Lead Sponsor	Collaborator
Wiktor Dega University Orthopedic and Rehabilitation Hospital

Country where clinical trial is conducted

Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	Functional assessment of patients using GMFM (Gross Motor Function Measure) scale		Through study completion, max. for 2.5 year every 6 months
Primary	X-ray or ultrasound scan imaging assessment of the spine, hip joints and bone mineral density		Once per 1 year
Primary	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	Hospitalization during or between rehabilitations that take place each 6 months		Through study completion, max. for 2.5 year
Secondary	Occurence of discomfort during therapy requiring abrupt interruption or significant modification		Through study completion, max. for 2.5 year every 6 months
Secondary	Number of fractures		Through study completion, max. for 2.5 year