An interventional study of Strength force measurment and Time tests in Muscular Atrophy, Spinal, Type II and Muscular Atrophy, Spinal, Type III, sponsored by Institut de Myologie, France. Withdrawn at 6 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-11-04.
Sponsored by Institut de Myologie, France · Not applicable, Interventional, and Other
funding issues
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.
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.
61 studies on the registry are indexed under Spinal Muscular Atrophies of Childhood; 18 are open to participants now.
Browse Spinal Muscular Atrophies of Childhood studies →Institut de Myologie, France is the lead sponsor of 30 studies on the registry; 5 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Non-Inclusion Criteria:
Exclusion criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients will be monitored and evaluate in a first time at home then onsite.
Other: Strength force measurment · Other: Time tests · Other: Motor scales · Other: Questionnaires · Device: Accelerometry · Other: MNR · Other: Bio-impedance analysis
Patients will be monitored and evaluate in a first time onsite then at home .
Other: Strength force measurment · Other: Time tests · Other: Motor scales · Other: Questionnaires · Device: Accelerometry · Other: MNR · Other: Bio-impedance analysis
The grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch)
Also known as: MyoGrip/Pinch
Lower and upper limb capacities of the patients will be measured during timed tests
Also known as: 10mWT, 30STS, 6MWT, 9HPT
Patient's motor functional abilities will be evaluated using specific motor scales (MFM32, RULM)
Patients and caregivers quality of life will be measured with different questionnaires (SMA-FRS, QOL-gNMD, SMAIS, PREM)
Patients physical activity will be measured at home using accelerometer sensors
Sub-group of patients will perform an NMR imaging to evaluate the intramuscular fatty infiltration in thighs and muscle volume in thighs
The patients' muscular and fatty volume will be evaluated using BIA technic (compared to MNR)
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
Time frame: Through study completion, an average of 3 weeks
To determine the barriers for evaluation at home
Inventory of items and assessments not carried out at home due to the environment
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Through study completion, an average of 3 weeks
Plan to share: Undecided
No publications or documents are linked to this record.
funding issues
funding issues
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is withdrawn, as verified in Nov 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Spinal Muscular Atrophies of Childhood →
Institut de Myologie, France