Muscular Atrophy, Spinal, Type IISMA-AtHomeNCT05839145
Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study
A study testing Strength force measurment, Time tests and Motor scales in people with Muscular Atrophy, Spinal, Type II and Muscular Atrophy, Spinal, Type III, run by Institut de Myologie, France at 6 sites in France.
Trial summary
There is no complete cure for SMA yet. However, the discovery of the genetic cause of SMA has led to the development of several treatment options that affect the genes involved in SMA - a gene replacement therapy called Zolgensma, and two drugs, called Nusinersen (Spinraza) and Risdiplam (Evyrsdi). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Muscular Atrophy, Spinal, Type II, Muscular Atrophy, Spinal, Type III
- What is being tested?
- Strength force measurment, Time tests and Motor scales
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 6 sitesFrance
- Study dates
- 6 Feb 2024to 1 Mar 2024
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age > 18 years
- Confirmed SMA type 2 or 3 diagnostic
- Written informed consent
- Able to comply with all protocol requirements
- Affiliate or beneficiary of a social security scheme
- Non-Inclusion Criteria:
Show all 10Show fewer
- Inability to carry out assessments at home
- Claustrophobia (only for patients from Paris and Lille sites)
- Guardianship/trusteeship
- Pregnant or nursing women
Who cannot join
- Inability to comply with protocol requirements
- Any medical and social conditions that could interfere with the study under the appreciation of the medical coordinator
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
France6 sites
CHU d'Angers
Angers, 49933
CHU de Lille
Lille, 59000
CHU de Nantes
Nantes, 44093
Institute of Myology
Paris, 75013
CHU de Reims
Reims, 51092
CHRU de Tours
Tours, 37044
About this study
There is no complete cure for SMA yet. However, the discovery of the genetic cause of SMA has led to the development of several treatment options that affect the genes involved in SMA - a gene replacement therapy called Zolgensma, and two drugs, called Nusinersen (Spinraza) and Risdiplam (Evyrsdi). In this context, the evaluation of efficacy and the long term follow-up of patients treated with these innovative treatments in clinical routine is one of the critical points. These evaluations are carried out in a medical context (clinical sites or research unit) using validated measurement tools and outcome measures. Carrying out these evaluations in a controlled environment can be considered from certain aspects as an advantage (reproducibility of measures, neutral environment, etc.), but also raises a certain number of questions regarding the impact on patients, the financial cost, or the relevance of the data obtained in an unnatural environment (stress, fatigue, patient motivation…). Also the regulatory authorities ask for longitudinal data for deciding to reimburse these expensive treatments. As such, the hospital cannot digest all these evaluations due to a lack of resources.
Read the detailed description
In the last few years, a number of therapeutic approaches have targeted a possible increase of the production of SMN protein in target motor neurons by genetic replacement of the defective SMN1 gene or by modifying pre-mRNA splicing in SMN2 to promote exon 7 inclusion by using an antisense oligonucleotide or small molecule drugs. Several clinical studies have focused on the evaluation of patients with SMA, whether they are ambulatory or not, adults, children or infants, treated or untreated.
Depending on the SMA type, age or ambulatory status of the patients, different assessments (motor function scales or questionnaires) have provided consistent results to measure the evolution of the patients, such as HFMSE, MFM, RULM, 6MWT, MRC scale, Chop Intend or HINE.
As these evaluations are generally carried out in a controlled environment, they are likely to present an environmental bias. Even if studies are designed to anticipate and avoid most of these issues, different factors can influence patient test results (fatigue, motivation, stress, day to day variability…). From an economical point of view, the evaluation of patients in a controlled environment also has a significant cost, which heavily impact the global cost of clinical research or standard care (transport, patients' accommodation and care…). This factor is even more important as a significant proportion of the SMA population is non-ambulatory.
New treatments are indicated to treat SMA with a major impact on pre-existing disease standards of care and patients care pathway. In particular, there is no consensus on appropriate measures to monitor disease progression and treatment effect in a real-world setting. Such measures are critically needed to discuss treatment indication (treatment initiation criteria and stopping rules, therapeutic goals) and treatment monitoring. While patient reported outcome measures (PROMs) become more represented, objective functional measures are still required to assess SMA. In spite of the development of digital measures, no validated patient self-reported functional measures can be used as a surrogate. Thus, the objective disease assessment is currently based on validated outcome measures for SMA, similar to those used in clinical studies. As compared to clinical trials, the feasibility to administer these measures to SMA patients is challenging. Major limiting factors are: (1) the high disease-prevalence, (2) time-consuming measures, (3) the need for trained expert evaluators, and (4) limited access to hospital-based resources. In addition, the burden of affected individuals and caregivers has not been evaluated as well as patient treatment monitoring expectations. A refined approach using modern tools and fitting with patient real life environment is needed.
- Being tested
- Strength force measurment, Time tests, Motor scales, Questionnaires, Accelerometry
- Keywords
- Muscular Atrophy, Spinal, Home monitoring, BioImpedance
- Medical subject headings
- Spinal Muscular Atrophies of Childhood, Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Strength force measurmentOther
Also called MyoGrip/Pinch
The grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch)
Time testsOther
Also called 10mWT, 30STS, 6MWT, 9HPT
Lower and upper limb capacities of the patients will be measured during timed tests
Motor scalesOther
Patient's motor functional abilities will be evaluated using specific motor scales (MFM32, RULM)
QuestionnairesOther
Patients and caregivers quality of life will be measured with different questionnaires (SMA-FRS, QOL-gNMD, SMAIS, PREM)
AccelerometryDevice
Patients physical activity will be measured at home using accelerometer sensors
MNROther
Sub-group of patients will perform an NMR imaging to evaluate the intramuscular fatty infiltration in thighs and muscle volume in thighs
Bio-impedance analysisOther
The patients' muscular and fatty volume will be evaluated using BIA technic (compared to MNR)
| Study group | Role | What they receive |
|---|---|---|
| Home to onsite monitoring | Other | Patients will be monitored and evaluate in a first time at home then onsite. Receives: Other: Strength force measurment; Other: Time tests; Other: Motor scales; Other: Questionnaires; Device: Accelerometry; Other: MNR; Other: Bio-impedance analysis |
| Onsite to Home monitoring | Other | Patients will be monitored and evaluate in a first time onsite then at home . Receives: Other: Strength force measurment; Other: Time tests; Other: Motor scales; Other: Questionnaires; Device: Accelerometry; Other: MNR; Other: Bio-impedance analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
To compare the results of physical evaluations between home and hospital
Evaluation of the correlations between results obtained during the physical evaluations at home compared to those obtained at hospital
Measured: Through study completion, an average of 3 weeks
Secondary outcomes (8)
To determine the barriers for evaluation at home
Inventory of items and assessments not carried out at home due to the environment
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital muscle volume measurement methods
Comparison of muscle volumes measured by bio-impedancemetry with those obtained by MRI.
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital MyoGrip measurement
Comparison of MyoGrip measurements obtained at home and at the hospital.
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital MyoPinch measurement
Comparison of MyoPinch measurements obtained at home and at the hospital.
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital MFM results
Comparison of MFM evaluation results obtained at home and at the hospital.
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital RULM results
Comparison of RULM evaluation results obtained at home and at the hospital.
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital 30STS tests results
Comparison of 30STS measurements obtained at home and at the hospital.
Measured: Through study completion, an average of 3 weeks
Correlation of home and hospital 9HPT results
Comparison of 9HPT measurements obtained at home and at the hospital.
Measured: Through study completion, an average of 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2023
- Study start6 Feb 2024
- Primary completion1 Mar 2024
- Study completion1 Mar 2024
Why it stopped: funding issues
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, Spinal, Type IIStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Collaborators
- Roche Pharma AG
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05839145
- Sponsor's protocol ID
- SMA-AtHome
- Acronym
- SMA-AtHome
- Status last verified
- Nov 2026
- First submitted
- 2 Dec 2022
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study
- ClinicalTrials.gov ID
- NCT05839145
- Acronym / sponsor ID
- SMA-AtHome / SMA-AtHome
- Keywords
- Muscular Atrophy, Spinal, Home monitoring, BioImpedance
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: funding issues
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Collaborators
- Roche Pharma AG
- Study start
- 6 Feb 2024 (actual)
- Primary completion
- 1 Mar 2024 (actual)
- Study completion
- 1 Mar 2024 (actual)
- First posted
- 3 May 2023 (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.
There is no complete cure for SMA yet. However, the discovery of the genetic cause of SMA has led to the development of several treatment options that affect the genes involved in SMA - a gene replacement therapy called Zolgensma, and two drugs, called Nusinersen (Spinraza) and Risdiplam (Evyrsdi). In this context, the evaluation of efficacy and the long term follow-up of patients treated with these innovative treatments in clinical routine is one of the critical points. These evaluations are carried out in a medical context (clinical sites or research unit) using validated measurement tools and outcome measures. Carrying out these evaluations in a controlled environment can be considered from certain aspects as an advantage (reproducibility of measures, neutral environment, etc.), but also raises a certain number of questions regarding the impact on patients, the financial cost, or the relevance of the data obtained in an unnatural environment (stress, fatigue, patient motivation…). Also the regulatory authorities ask for longitudinal data for deciding to reimburse these expensive treatments. As such, the hospital cannot digest all these evaluations due to a lack of resources.
Detailed description
In the last few years, a number of therapeutic approaches have targeted a possible increase of the production of SMN protein in target motor neurons by genetic replacement of the defective SMN1 gene or by modifying pre-mRNA splicing in SMN2 to promote exon 7 inclusion by using an antisense oligonucleotide or small molecule drugs. Several clinical studies have focused on the evaluation of patients with SMA, whether they are ambulatory or not, adults, children or infants, treated or untreated.
Depending on the SMA type, age or ambulatory status of the patients, different assessments (motor function scales or questionnaires) have provided consistent results to measure the evolution of the patients, such as HFMSE, MFM, RULM, 6MWT, MRC scale, Chop Intend or HINE.
As these evaluations are generally carried out in a controlled environment, they are likely to present an environmental bias. Even if studies are designed to anticipate and avoid most of these issues, different factors can influence patient test results (fatigue, motivation, stress, day to day variability…). From an economical point of view, the evaluation of patients in a controlled environment also has a significant cost, which heavily impact the global cost of clinical research or standard care (transport, patients' accommodation and care…). This factor is even more important as a significant proportion of the SMA population is non-ambulatory.
New treatments are indicated to treat SMA with a major impact on pre-existing disease standards of care and patients care pathway. In particular, there is no consensus on appropriate measures to monitor disease progression and treatment effect in a real-world setting. Such measures are critically needed to discuss treatment indication (treatment initiation criteria and stopping rules, therapeutic goals) and treatment monitoring. While patient reported outcome measures (PROMs) become more represented, objective functional measures are still required to assess SMA. In spite of the development of digital measures, no validated patient self-reported functional measures can be used as a surrogate. Thus, the objective disease assessment is currently based on validated outcome measures for SMA, similar to those used in clinical studies. As compared to clinical trials, the feasibility to administer these measures to SMA patients is challenging. Major limiting factors are: (1) the high disease-prevalence, (2) time-consuming measures, (3) the need for trained expert evaluators, and (4) limited access to hospital-based resources. In addition, the burden of affected individuals and caregivers has not been evaluated as well as patient treatment monitoring expectations. A refined approach using modern tools and fitting with patient real life environment is needed.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Home to onsite monitoring | Other | Other: Strength force measurment, Other: Time tests, Other: Motor scales, Other: Questionnaires, Device: Accelerometry, Other: MNR, Other: Bio-impedance analysisPatients will be monitored and evaluate in a first time at home then onsite. |
| Onsite to Home monitoring | Other | Other: Strength force measurment, Other: Time tests, Other: Motor scales, Other: Questionnaires, Device: Accelerometry, Other: MNR, Other: Bio-impedance analysisPatients will be monitored and evaluate in a first time onsite then at home . |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age > 18 years
- Confirmed SMA type 2 or 3 diagnostic
- Written informed consent
- Able to comply with all protocol requirements
- Affiliate or beneficiary of a social security scheme
Key exclusion criteria (2 listed)
- Inability to comply with protocol requirements
- Any medical and social conditions that could interfere with the study under the appreciation of the medical coordinator
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 |
|---|---|---|
| Strength force measurmentAlso: MyoGrip/PinchThe grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch) | Other | Home to onsite monitoring, Onsite to Home monitoring |
| Time testsAlso: 10mWT, 30STS, 6MWT, 9HPTLower and upper limb capacities of the patients will be measured during timed tests | Other | Home to onsite monitoring, Onsite to Home monitoring |
| Motor scalesPatient's motor functional abilities will be evaluated using specific motor scales (MFM32, RULM) | Other | Home to onsite monitoring, Onsite to Home monitoring |
| QuestionnairesPatients and caregivers quality of life will be measured with different questionnaires (SMA-FRS, QOL-gNMD, SMAIS, PREM) | Other | Home to onsite monitoring, Onsite to Home monitoring |
| AccelerometryPatients physical activity will be measured at home using accelerometer sensors | Device | Home to onsite monitoring, Onsite to Home monitoring |
| MNRSub-group of patients will perform an NMR imaging to evaluate the intramuscular fatty infiltration in thighs and muscle volume in thighs | Other | Home to onsite monitoring, Onsite to Home monitoring |
| Bio-impedance analysisThe patients' muscular and fatty volume will be evaluated using BIA technic (compared to MNR) | Other | Home to onsite monitoring, Onsite to Home monitoring |
Primary outcome
To compare the results of physical evaluations between home and hospital
Time frame: Through study completion, an average of 3 weeks
Evaluation of the correlations between results obtained during the physical evaluations at home compared to those obtained at hospital
8 secondary outcomes
To determine the barriers for evaluation at home
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital muscle volume measurement methods
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital MyoGrip measurement
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital MyoPinch measurement
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital MFM results
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital RULM results
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital 30STS tests results
Time frame: Through study completion, an average of 3 weeks
Correlation of home and hospital 9HPT results
Time frame: Through study completion, an average of 3 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Strength force measurment, Time tests and Motor scales in people with Muscular Atrophy, Spinal, Type II and Muscular Atrophy, Spinal, Type III, run by Institut de Myologie, France at 6 sites in France.
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 10 inclusion and 2 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: withdrawn, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
To compare the results of physical evaluations between home and hospital (time frame: Through study completion, an average of 3 weeks). 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 4 Nov 2026; AzTrials last refreshed its copy on 8 Nov 2026.