- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04833348
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies (IMUSMA)
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies, IMUSMA Project
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Infantile spinal muscular atrophy is a common disease (the second most common fatal autosomal recessive disease after cystic fibrosis), neurodegenerative disorders of childhood causing severe motor impairment and a risk to life through respiratory failure in the most severe forms.
Innovative therapies (gene therapy or pharmacogenetics) have recently proven their effectiveness on survival criteria. Nevertheless, the motor benefit of these therapies must be evaluated more precisely.
Currently, the reference methods for motor development assessment are fairly robust semi-quantitative motor scales that lack sensitivity and do not reflect function (CHOPINTEND, HINE, BAYLEY SCALE, MFM and CGI-scale).
Advances in recent techniques have enabled the emergence of non-invasive, secure, easy-to-use inertial sensors in routine clinical practice that allow quantification of infant movements.
The aim of the study is to propose a method for quantifying motor function in infants with spinal muscular atrophy treated with innovative therapies using inertial sensors.
Study Type
Enrollment (Estimated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Isabelle DESGUERRE, MD, PhD
- Phone Number: +33 1 44 49 48 56
- Email: isabelle.desguerre@aphp.fr
Study Contact Backup
- Name: Hélène Morel
- Phone Number: +33 1 71 19 63 46
- Email: helene.morel@aphp.fr
Study Locations
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-
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Paris, France, 75015
- Recruiting
- Hôpital Necker-Enfants Malades
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Contact:
- Isabelle DESGUERRE, MD, PhD
- Phone Number: +33 1 44 49 48 56
- Email: isabelle.desguerre@aphp.fr
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Sub-Investigator:
- Rémi BARROIS, PhD
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Sub-Investigator:
- Christine BARNERIAS, MD
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Infants of both sexes
- Suffering from spinal muscular atrophy (diagnosis by genetic study "homozygous deletion of SMN1")
- Followed up by the Necker Neuromuscular Reference Center (GNMH)
Eligible for innovative therapy (gene therapy or pharmacogenetics)
- age of onset of the disease <1 year
- no severe respiratory impairment (dependence on ventilatory support for more than 16 hours per day) or bulbar involvement
- decision of treatment by a Multidisciplinary Consultation Meeting national of experts
- Benefiting from social security scheme
- Informed consent signed by holders of parental authority and the investigator
Exclusion Criteria:
- Non-consent of one of the holders of parental authority
- Respiratory instability (dependence on ventilatory support for more than 16 hours per day) or hemodynamics
- Contraindication to innovative therapy
- History of another disease impacting motor skills (neonatal suffering, etc.)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Patients
Infants with spinal muscular atrophy cared by the Neuromuscular Reference Center at Necker Hospital and eligible for innovative therapy (gene therapy or pharmacogenetics)
|
Measurement of motor skills at M0: start of the administration of the innovative therapy and then 1 month, 3 months, 6 months, 1 year and then 2 years later:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in the 95th percentile of the norm of acceleration
Time Frame: Month 0 to month 24
|
95th percentile of the norm of the acceleration of the feet and the arms.
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Month 0 to month 24
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in the 95th percentile of the norm of angular velocity
Time Frame: Month 0 to month 24
|
95th percentile of the norm of angular velocity of the feet and the arms.
|
Month 0 to month 24
|
Change in the 95th percentile of the accelerations allong the vertical axis and the horizontal plane
Time Frame: Month 0 to month 24
|
95th percentile of the accelerations of the feet and the arms the vertical axis and the horizontal plane.
|
Month 0 to month 24
|
Change in the 95th percentile of the angular velocities allong the vertical axis and the horizontal plane.
Time Frame: Month 0 to month 24
|
95th percentile of the angular velocities of the feet and the arms the vertical axis and the horizontal plane.
|
Month 0 to month 24
|
Change in the acceleration's entropy
Time Frame: Month 0 to month 24
|
Acceleration's entropy computed in the different axis of the feet and the arms.
|
Month 0 to month 24
|
Collaborators and Investigators
Investigators
- Principal Investigator: Isabelle DESGUERRE, MD, PhD, Assistance Publique - Hôpitaux de Paris
- Study Director: Brian TERVIL, PhD, Centre Borelli - Université Paris Descartes
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- APHP201640
- 2020-A02279-30 (Other Identifier: ID-RCB Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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