Spinal Muscular AtrophyNusiMFMNCT04602195
Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
An observational study of Spinal Muscular Atrophy, run by Hospices Civils de Lyon at 10 sites in France.
Trial summary
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole… Read more about this study
- Who is it for?
- Ages 2 to 6 years
- Conditions the study is for
- Spinal Muscular Atrophy
- How many people
- 33 participants
- Where
- 10 sitesFrance
- Study dates
- 19 Jan 2021to 10 Nov 2023
- Phase
- Observational
- 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 2 to 6 years
- All sexes
- No healthy volunteers
- Child
Study population: * Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed * Aged 2 to 6 years old * Treated by Nusinersen for at least two months * With parental assent
Who may be able to join
- Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed
- Aged 2 to 6 years old
- Treated by Nusinersen for at least two months
- With parental assent
Who cannot join
- Patients with associated cognitive impairment making impossible evaluation of motor function
- Patients participating to a clinical study with a potential effect on their motor function.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
France10 sites
Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence des Maladies Neuromusculaires AOC CHU d'Angers
Angers, 49933
Centre de Référence des maladies neuromusculaires Centre Hospitalier Régional Universitaire de Brest
Brest, 29200
L'Escale, Service central de rééducation pédiatrique Hospices Civils de Lyon Hôpital Mere Enfant
Bron, 69677
Service de génétique médicale Pôle de Pédiatrie CHU Estaing
Clermont-Ferrand, 63003
Service de Neurologie et réanimation pédiatriques
Garches, 92380
Apf Esean
Nantes, 44200
Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires
Paris, 75571
Service de pédiatrie CHU de Saint-Etienne
Saint-Priest-en-Jarez, 42270
Service de Neuropédiatrie CHU de Strasbourg - Hôpital de Hautepierre
Strasbourg, 67200
Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse
Toulouse, 31059
About this study
Official title: Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen: NusiMFM
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items originally validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items originally validated for patients between 2 and 6 years old (MFM-20).
In order to prove the possible use of MFM-32 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-32 and the validity of the scale in this population.
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, Motor Function Measure,Nusinersen
- Medical subject headings
- Muscular Atrophy, Spinal
What is being measured
The primary outcome is the main question the study is designed to answer.
MFM-32 responsiveness
Responsiveness of MFM-32 in comparison to PGIC. The responsiveness sensitivity to change indexes considered will be the effect size (ES) amplitude and the Standardized Response Mean (SRM)
Measured: 1 Year after the first MFM-32 assessment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2020
- Study start19 Jan 2021
- Primary completion10 Nov 2023
- Study completion10 Nov 2023
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.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Laure Le Goff, MD, Principal investigator, L'Escale, Service central de rééducation pédiatrique
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04602195
- Sponsor's protocol ID
- 69HCL19_0875
- Acronym
- NusiMFM
- Status last verified
- Apr 2023
- First submitted
- 20 Oct 2020
- Last updated
- 21 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen: NusiMFM
- ClinicalTrials.gov ID
- NCT04602195
- Acronym / sponsor ID
- NusiMFM / 69HCL19_0875
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, Motor Function Measure,Nusinersen
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 19 Jan 2021 (actual)
- Primary completion
- 10 Nov 2023 (actual)
- Study completion
- 10 Nov 2023 (actual)
- First posted
- 26 Oct 2020 (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.
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items originally validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items originally validated for patients between 2 and 6 years old (MFM-20).
In order to prove the possible use of MFM-32 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-32 and the validity of the scale in this population.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- * Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed * Aged 2 to 6 years old * Treated by Nusinersen for at least two months * With parental assent
Key inclusion criteria (4 listed)
- Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed
- Aged 2 to 6 years old
- Treated by Nusinersen for at least two months
- With parental assent
Key exclusion criteria (2 listed)
- Patients with associated cognitive impairment making impossible evaluation of motor function
- Patients participating to a clinical study with a potential effect on their motor function.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
MFM-32 responsiveness
Time frame: 1 Year after the first MFM-32 assessment
Responsiveness of MFM-32 in comparison to PGIC. The responsiveness sensitivity to change indexes considered will be the effect size (ES) amplitude and the Standardized Response Mean (SRM)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2023.
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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.
Laure Le Goff, MD principal investigator
L'Escale, Service central de rééducation pédiatrique
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinal Muscular Atrophy, run by Hospices Civils de Lyon at 10 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 4 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: finished, as of the sponsor's last update on 21 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
MFM-32 responsiveness (time frame: 1 Year after the first MFM-32 assessment). 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 Dec 2023.