SMASMArtNCT06322654
A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients
A study testing Robotically Assisted Locomotion and Robotically Assisted Verticalization in people with SMA, run by Wiktor Dega University Orthopedic and Rehabilitation Hospital at 1 site in Poland.
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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- SMA
- What is being tested?
- Robotically Assisted Locomotion and Robotically Assisted Verticalization
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 sitePoland
- Study dates
- 21 Dec 2023to Jul 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Poland1 site, 1 recruiting
- Recruiting
Wiktor Dega University Orthopedic and Rehabilitation Hospital
Poznan, 61-545
Marek Jóźwiak, 696052475 ext. +48, sma@orsk.pl
Radosław Rutkowski, 696052475, sma@orsk.pl
About this study
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
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:
Read more
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.
- Research period: 4 years
- Patients age: 0-21 y.o.
- Group size: 200 patients (100 patients in each group)
- Assignment of patients to study groups in a randomised manner
- Conditions
- SMA
- Being tested
- Robotically Assisted Locomotion, Robotically Assisted Verticalization
- Keywords
- SMA, Robotically-assisted therapy, Robotically Assisted Locomotion, Robotically Assisted Verticalization
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Robotically Assisted LocomotionProcedure or surgery
- 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 VerticalizationProcedure or surgery
- Motor control training
- Verticalization training with Standard Frame Devices
- Locomotion and motor control training with elements of balance and coordination
- Cognitive therapy
| Study group | Role | What they receive |
|---|---|---|
| Robotically Assisted Locomotion | Experimental | Not described Receives: Procedure: Robotically Assisted Locomotion |
| Robotically Assisted Verticalization | Active comparator | Not described Receives: Procedure: Robotically Assisted Verticalization |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale
Measured: 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.
Measured: Through study completion, max. for 2.5 year every 6 months
Functional assessment of patients using GMFM (Gross Motor Function Measure) scale
Measured: 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
Measured: Once per 1 year
Assessment of the quality of life of patients and their caregivers using Pediatric Quality of Life Inventory ver. 4.0
Measured: 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
Measured: Through study completion, max. for 2.5 year
Occurence of discomfort during therapy requiring abrupt interruption or significant modification
Measured: Through study completion, max. for 2.5 year every 6 months
Number of fractures
Measured: Through study completion, max. for 2.5 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2024
- Study start21 Dec 2023
- Primary completion31 Mar 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SMAStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- There are two research groups with 100 patients in each group. 1. Experimental: Robotically Assisted Locomotion 2. Comparison: Robotically Assisted Verticalization
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wiktor Dega University Orthopedic and Rehabilitation Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Marek Jóźwiak, Principal investigator, Wiktor Dega University Orthopedic and Rehabilitation Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06322654
- Sponsor's protocol ID
- 2023/ABM/01/00004/P/02
- Acronym
- SMArt
- Status last verified
- Mar 2024
- First submitted
- 26 Jan 2024
- Last updated
- 21 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06322654
- Acronym / sponsor ID
- SMArt / 2023/ABM/01/00004/P/02
- Conditions
- SMA
- Keywords
- SMA, Robotically-assisted therapy, Robotically Assisted Locomotion, Robotically Assisted Verticalization
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Wiktor Dega University Orthopedic and Rehabilitation HospitalUniversity, hospital or other
- Study start
- 21 Dec 2023 (actual)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 21 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
- Research period: 4 years
- Patients age: 0-21 y.o.
- Group size: 200 patients (100 patients in each group)
- Assignment of patients to study groups in a randomised manner
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
- Model description
- There are two research groups with 100 patients in each group. 1. Experimental: Robotically Assisted Locomotion 2. Comparison: Robotically Assisted Verticalization
| Arm | Type | Receives |
|---|---|---|
| Robotically Assisted Locomotion | Experimental | Procedure: Robotically Assisted Locomotion |
| Robotically Assisted Verticalization | Active comparator | Procedure: Robotically Assisted Verticalization |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Procedure or surgery | Robotically Assisted Locomotion |
| 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 | Procedure or surgery | Robotically Assisted Verticalization |
Primary outcome
Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale
Time frame: 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.
Time frame: Through study completion, max. for 2.5 year every 6 months
Functional assessment of patients using GMFM (Gross Motor Function Measure) scale
Time frame: 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
Time frame: Once per 1 year
Assessment of the quality of life of patients and their caregivers using Pediatric Quality of Life Inventory ver. 4.0
Time frame: Through study completion, max. for 2.5 year every 6 months
3 secondary outcomes
Hospitalization during or between rehabilitations that take place each 6 months
Time frame: Through study completion, max. for 2.5 year
Occurence of discomfort during therapy requiring abrupt interruption or significant modification
Time frame: Through study completion, max. for 2.5 year every 6 months
Number of fractures
Time frame: Through study completion, max. for 2.5 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Marek Jóźwiak principal investigator
Wiktor Dega University Orthopedic and Rehabilitation Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Robotically Assisted Locomotion and Robotically Assisted Verticalization in people with SMA, run by Wiktor Dega University Orthopedic and Rehabilitation Hospital at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Marek Jóźwiak (696052475 ext. +48, sma@orsk.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale (time frame: Through study completion, max. for 2.5 year every 6 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2024.