Spinal Muscular AtrophyNCT04888702
Holter of Movement in Patients with SMA Undergoing Treatment.
A study testing Actimyo in people with Spinal Muscular Atrophy, run by Centre Hospitalier Universitaire de Liege at 3 sites in 2 countries.
Trial summary
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Actimyo
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 8 Aug 2018to 30 May 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically confirmed spinal muscular atrophy.
- Treatment by Spinraza ° or by risdiplam planned.
- Over 6 years old.
- Signed informed consent.
Who cannot join
- Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Belgium2 sites
Erasme Hospital - Cliniques universitaires de Bruxelles
Brussels, 1000
Centre Hospitalier Régional de la Citadelle
Liège, Liège, 4000
Romania1 site
National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu"
Bucharest
About this study
Official title: Holter of Movement in Patients with Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment with Spinraza ° or Risdiplam. Acti-SMA Study.
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. A secondary objective would also be to identify suitable accelerometric measurements that are sensitive to change but also well correlated to other clinical scores.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- Actimyo
- Keywords
- Actimyo, Accelerometry, Motor neuron disease, Risdiplam, Nusinersen, Home monitoring
- Medical subject headings
- Muscular Atrophy, Spinal, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ActimyoDevice
Actimyo° is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle.
| Study group | Role | What they receive |
|---|---|---|
| SMA patients | Experimental | Patients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment. Receives: Device: Actimyo |
What is being measured
The primary outcome is the main question the study is designed to answer.
95th centile of stride velocity
95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life.
(meter per second).
Measured: 39 months
50th centile of stride velocity
50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
Measured: 39 months
95th centile of stride length
95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Measured: 39 months
50th centile of stride length
50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Measured: 39 months
Upper limb function evolution in patient with SMA undergoing treatment
Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.
Measured: 39 months
Secondary outcomes (8)
6-minute walk test
The Six-Minute Walk Test is a functional walking test in which investigators assess the distance the patient can walk in six minutes (meter).
Measured: 39 months
Dynamometric measures
Assessment of upper limb strength with myogrip and myopinch (kg).
Measured: 39 months
10-metre walk test
The 10-metre walk test is a functional walking test in which investigators assess the time the patient take to walk 10 meters (second).
Measured: 39 months
rise from floor
The rise from floor is a functional test in which investigators assess the time the patient take to rise from the floor (second).
Measured: 39 months
4-stair climb
The 4-stair climbr is a functional test in which investigators assess the time the patient take to climb a four-step staircase (second).
Measured: 39 months
revised upper limb module (RULM)
Revised Upper Limb Module (RULM) is an assessment specifically designed for upper limb function in Spinal Muscular Atrophy.
Measured: 39 months
Hamersmith funcitonnal motor scale (HFMS)
HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy.
Measured: 39 months
motor function measure (MFM)
Motor Function Measure is a global quantitative scale created to measure the functional motor abilities of a person affected by a neuromuscular disease.
Measured: 39 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2021
- Study start8 Aug 2018
- Primary completion30 Jan 2025
- Study completion30 May 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- all patients undergo clinical evaluation and wear Actimyo device in daily living to evaluate their improvement under treatment.
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Liege
- Sponsor type
- University, hospital or other
- Collaborators
- SYSNAV, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Régional de la Citadelle
- Responsible party
- Principal investigator, Margaux POLEUR, Centre Hospitalier Universitaire de Liege
- Investigators
- Margaux Poleur, Principal investigator, CHR Citadelle-CHU liège
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04888702
- Sponsor's protocol ID
- ActiSMA.
- Status last verified
- May 2024
- First submitted
- 11 Mar 2021
- Last updated
- 12 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Holter of Movement in Patients with Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment with Spinraza ° or Risdiplam. Acti-SMA Study.
- ClinicalTrials.gov ID
- NCT04888702
- Acronym / sponsor ID
- ActiSMA.
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Actimyo, Accelerometry, Motor neuron disease, Risdiplam, Nusinersen, Home monitoring
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de LiegeUniversity, hospital or other
- Collaborators
- SYSNAV, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Régional de la Citadelle
- Study start
- 8 Aug 2018 (actual)
- Primary completion
- 30 Jan 2025 (actual)
- Study completion
- 30 May 2025 (estimated)
- First posted
- 17 May 2021 (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.
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. A secondary objective would also be to identify suitable accelerometric measurements that are sensitive to change but also well correlated to other clinical scores.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- all patients undergo clinical evaluation and wear Actimyo device in daily living to evaluate their improvement under treatment.
| Arm | Type | Receives |
|---|---|---|
| SMA patients | Experimental | Device: ActimyoPatients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Genetically confirmed spinal muscular atrophy.
- Treatment by Spinraza ° or by risdiplam planned.
- Over 6 years old.
- Signed informed consent.
Key exclusion criteria (1 listed)
- Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.
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 |
|---|---|---|
| ActimyoActimyo° is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle. | Device | SMA patients |
Primary outcome
95th centile of stride velocity
Time frame: 39 months
95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life. (meter per second).
50th centile of stride velocity
Time frame: 39 months
50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
95th centile of stride length
Time frame: 39 months
95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
50th centile of stride length
Time frame: 39 months
50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Upper limb function evolution in patient with SMA undergoing treatment
Time frame: 39 months
Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.
8 secondary outcomes
6-minute walk test
Time frame: 39 months
Dynamometric measures
Time frame: 39 months
10-metre walk test
Time frame: 39 months
rise from floor
Time frame: 39 months
4-stair climb
Time frame: 39 months
revised upper limb module (RULM)
Time frame: 39 months
Hamersmith funcitonnal motor scale (HFMS)
Time frame: 39 months
motor function measure (MFM)
Time frame: 39 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2025.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Margaux Poleur principal investigator
CHR Citadelle-CHU liège
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Actimyo in people with Spinal Muscular Atrophy, run by Centre Hospitalier Universitaire de Liege at 3 sites in 2 countries.
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 1 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: status not confirmed, as of the sponsor's last update on 12 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Belgium, Romania. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
95th centile of stride velocity (time frame: 39 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 12 Feb 2025.