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Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies (IMUSMA)

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ClinicalTrials.gov Identifier: NCT04833348
Recruitment Status : Not yet recruiting
First Posted : April 6, 2021
Last Update Posted : June 29, 2021
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Other: Motor function measurement using inertial sensors Not Applicable

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies, IMUSMA Project
Estimated Study Start Date : August 2021
Estimated Primary Completion Date : August 2025
Estimated Study Completion Date : August 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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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)
 Other: Motor function measurement using inertial sensors

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:

  • Free motor skills in the supine position
  • Motricity in the supine position stimulated by a play frame
  • Measurement of the proximal and distal activity of the upper limbs in motor skills stimulated by the play gantry
  • Measurement of activity in a supported sitting position Longitudinal study, the subject is his own control


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.


Eligibility Criteria
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Ages Eligible for Study:   up to 2 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04833348


Contacts
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Contact: Isabelle DESGUERRE, MD, PhD 1 44 49 48 56 ext +33 isabelle.desguerre@aphp.fr
Contact: Hélène MOREL 1 71 19 63 46 ext +33 helene.morel@aphp.fr

Locations
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France
Hôpital Necker-Enfants Malades
Paris, France, 75015
Contact: Isabelle DESGUERRE, MD, PhD    1 44 49 48 56 ext +33    isabelle.desguerre@aphp.fr   
Sub-Investigator: Rémi BARROIS, PhD         
Sub-Investigator: Christine BARNERIAS, MD         
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
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Principal Investigator: Isabelle DESGUERRE, MD, PhD Assistance Publique - Hôpitaux de Paris
Study Director: Brian TERVIL, PhD Centre Borelli - Université Paris Descartes
More Information
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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT04833348    
Other Study ID Numbers: APHP201640
2020-A02279-30 ( Other Identifier: ID-RCB Number )
First Posted: April 6, 2021    Key Record Dates
Last Update Posted: June 29, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Assistance Publique - Hôpitaux de Paris:
Spinal muscular atrophy
Innovative therapies
Motor function
Wearable inertial sensors
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
 Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases