Spinal Muscular AtrophyRetroNusiMFMNCT04644393
Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen
An observational study of Spinal Muscular Atrophy, run by Hospices Civils de Lyon at 8 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
- 40 participants
- Where
- 8 sitesFrance
- Study dates
- Nov 2020to Mar 2021
- 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 at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 months
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 at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 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
8 sites in 1 country.
8 sites in 1 country
France8 sites
Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence maladies neuromusculaires AOC CHU d'Angers
Angers
Julien DURIGNEUX, MD, 02 41 35 44 45, judurigneux@chu-angers.fr
Service de génétique médicale Pôle de Pédiatrie CHU Estaing
Clermont-Ferrand
Catherine SARRET, MD, 04.73.75.06.53;, csarret@chu-clermontferrand.fr
Service de Neurologie et réanimation pédiatriques CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Garche
Susana QUIJANO-ROY, MD, 01.47.41.78.90;, susana.quijano-y@aphp.fr
Hospices Civils de Lyon, Hôpital Femme Mère Enfant
Lyon
Laure LE GOFF, MD, +33 4 72 12 95 04, laure.le-goff@chu-lyon.fr
Laure LE GOFF, MD
Apf Esean
Nantes
Capucine de LATTRE, MD, 02 40 34 76 74, c.delattre@esean.fr
Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires CHU Paris Est - Hôpital d'Enfants Armand-Trousseau
Paris
Andreea SEFERIAN, MD, 01 44 73 65 37, a.seferian@institut-myologie.org
Service de pédiatrie CHU de Saint-Etienne
Saint-Priest-en-Jarez
Gaelle Gousse, MD, 04 77 82 87 29, gaelle.gousse@chu-st-etienne.fr
Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse
Toulouse
Claude CANCES, MD, 05.34.55.85.77;, cances.c@chu-toulouse.fr
About this study
Official title: Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen: Retro 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 validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items validated for patients between 2 and 6 years old (MFM-20).
In order to show the possible use of MFM-20 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-20 and the validity of the scale in this population.
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, Motor Function Measure, Outcome Assessment, 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-20 responsiveness
The MFM-20 sensitivity to change indexes considered will be the effect amplitude and the Standardized Response Mean (SRM).
Measured: At least 6 Months after the first MFM-20 assessment, and at maximum 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2020
- Study startNov 2020 (estimated)
- Primary completionMar 2021 (estimated)
- Study completionMar 2021 (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.
- 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
- Retrospective
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04644393
- Sponsor's protocol ID
- 69HCL20_1086
- Acronym
- RetroNusiMFM
- Status last verified
- Nov 2020
- First submitted
- 20 Nov 2020
- Last updated
- 25 Nov 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen: Retro NusiMFM
- ClinicalTrials.gov ID
- NCT04644393
- Acronym / sponsor ID
- RetroNusiMFM / 69HCL20_1086
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, Motor Function Measure, Outcome Assessment, Nusinersen
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- Nov 2020 (estimated)
- Primary completion
- Mar 2021 (estimated)
- Study completion
- Mar 2021 (estimated)
- First posted
- 25 Nov 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 validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items validated for patients between 2 and 6 years old (MFM-20).
In order to show the possible use of MFM-20 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-20 and the validity of the scale in this population.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
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 at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 months
Key inclusion criteria (5 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 at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 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-20 responsiveness
Time frame: At least 6 Months after the first MFM-20 assessment, and at maximum 24 months
The MFM-20 sensitivity to change indexes considered will be the effect amplitude and the Standardized Response Mean (SRM).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2020.
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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.
8 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 8 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 5 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: status not confirmed, as of the sponsor's last update on 25 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Laure LE GOFF, MD (+33 4 72 12 95 04, laure.le-goff@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MFM-20 responsiveness (time frame: At least 6 Months after the first MFM-20 assessment, and at maximum 24 months). 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 25 Nov 2020.