Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study
NCT ID: NCT05839145
Last Updated: 2023-12-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
NA
60 participants
INTERVENTIONAL
2023-12-15
2024-01-31
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
OTHER
NONE
Study Groups
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Home to onsite monitoring
Patients will be monitored and evaluate in a first time at home then onsite.
Strength force measurment
The grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch)
Time tests
Lower and upper limb capacities of the patients will be measured during timed tests
Motor scales
Patient's motor functional abilities will be evaluated using specific motor scales (MFM32, RULM)
Questionnaires
Patients and caregivers quality of life will be measured with different questionnaires (SMA-FRS, QOL-gNMD, SMAIS, PREM)
Accelerometry
Patients physical activity will be measured at home using accelerometer sensors
MNR
Sub-group of patients will perform an NMR imaging to evaluate the intramuscular fatty infiltration in thighs and muscle volume in thighs
Bio-impedance analysis
The patients' muscular and fatty volume will be evaluated using BIA technic (compared to MNR)
Onsite to Home monitoring
Patients will be monitored and evaluate in a first time onsite then at home .
Strength force measurment
The grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch)
Time tests
Lower and upper limb capacities of the patients will be measured during timed tests
Motor scales
Patient's motor functional abilities will be evaluated using specific motor scales (MFM32, RULM)
Questionnaires
Patients and caregivers quality of life will be measured with different questionnaires (SMA-FRS, QOL-gNMD, SMAIS, PREM)
Accelerometry
Patients physical activity will be measured at home using accelerometer sensors
MNR
Sub-group of patients will perform an NMR imaging to evaluate the intramuscular fatty infiltration in thighs and muscle volume in thighs
Bio-impedance analysis
The patients' muscular and fatty volume will be evaluated using BIA technic (compared to MNR)
Interventions
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Strength force measurment
The grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch)
Time tests
Lower and upper limb capacities of the patients will be measured during timed tests
Motor scales
Patient's motor functional abilities will be evaluated using specific motor scales (MFM32, RULM)
Questionnaires
Patients and caregivers quality of life will be measured with different questionnaires (SMA-FRS, QOL-gNMD, SMAIS, PREM)
Accelerometry
Patients physical activity will be measured at home using accelerometer sensors
MNR
Sub-group of patients will perform an NMR imaging to evaluate the intramuscular fatty infiltration in thighs and muscle volume in thighs
Bio-impedance analysis
The patients' muscular and fatty volume will be evaluated using BIA technic (compared to MNR)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* Inability to carry out assessments at home
* Claustrophobia (only for patients from Paris and Lille sites)
* Guardianship/trusteeship
* Pregnant or nursing women
Exclusion Criteria
* Any medical and social conditions that could interfere with the study under the appreciation of the medical coordinator
18 Years
ALL
No
Sponsors
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Roche Pharma AG
INDUSTRY
Institut de Myologie, France
OTHER
Responsible Party
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Locations
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CHU d'Angers
Angers, , France
CHU de Lille
Lille, , France
CHU de Nantes
Nantes, , France
Institute of Myology
Paris, , France
CHU de Reims
Reims, , France
CHRU de Tours
Tours, , France
Countries
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Central Contacts
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Facility Contacts
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Marco Spinazzi, MD
Role: primary
Céline Tard, MD
Role: primary
Yann Pereon, MD
Role: primary
Guillaume Bassez, MD
Role: primary
François Boyer, Pr
Role: primary
Sybille Pellieux, MD
Role: primary
Other Identifiers
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SMA-AtHome
Identifier Type: -
Identifier Source: org_study_id