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

Primary Purpose

Spinal Muscular Atrophy

Status
Recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Motor function measurement using inertial sensors
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Spinal Muscular Atrophy focused on measuring Spinal muscular atrophy, Innovative therapies, Motor function, Wearable inertial sensors

Eligibility Criteria

undefined - 2 Years (Child)All SexesDoes not accept healthy volunteers

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.)

Sites / Locations

  • Hôpital Necker-Enfants MaladesRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Patients

Arm Description

Infants with spinal muscular atrophy cared by the Neuromuscular Reference Center at Necker Hospital and eligible for innovative therapy (gene therapy or pharmacogenetics)

Outcomes

Primary Outcome Measures

Change in the 95th percentile of the norm of acceleration
95th percentile of the norm of the acceleration of the feet and the arms.

Secondary Outcome Measures

Change in the 95th percentile of the norm of angular velocity
95th percentile of the norm of angular velocity of the feet and the arms.
Change in the 95th percentile of the accelerations allong the vertical axis and the horizontal plane
95th percentile of the accelerations of the feet and the arms the vertical axis and the horizontal plane.
Change in the 95th percentile of the angular velocities allong the vertical axis and the horizontal plane.
95th percentile of the angular velocities of the feet and the arms the vertical axis and the horizontal plane.
Change in the acceleration's entropy
Acceleration's entropy computed in the different axis of the feet and the arms.

Full Information

First Posted
March 23, 2021
Last Updated
October 10, 2022
Sponsor
Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT04833348
Brief Title
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
Acronym
IMUSMA
Official Title
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies, IMUSMA Project
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
March 20, 2021 (Actual)
Primary Completion Date
March 2025 (Anticipated)
Study Completion Date
March 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy
Keywords
Spinal muscular atrophy, Innovative therapies, Motor function, Wearable inertial sensors

7. Study Design

Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Patients
Arm Type
Experimental
Arm Description
Infants with spinal muscular atrophy cared by the Neuromuscular Reference Center at Necker Hospital and eligible for innovative therapy (gene therapy or pharmacogenetics)
Intervention Type
Other
Intervention Name(s)
Motor function measurement using inertial sensors
Intervention Description
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
Primary Outcome Measure Information:
Title
Change in the 95th percentile of the norm of acceleration
Description
95th percentile of the norm of the acceleration of the feet and the arms.
Time Frame
Month 0 to month 24
Secondary Outcome Measure Information:
Title
Change in the 95th percentile of the norm of angular velocity
Description
95th percentile of the norm of angular velocity of the feet and the arms.
Time Frame
Month 0 to month 24
Title
Change in the 95th percentile of the accelerations allong the vertical axis and the horizontal plane
Description
95th percentile of the accelerations of the feet and the arms the vertical axis and the horizontal plane.
Time Frame
Month 0 to month 24
Title
Change in the 95th percentile of the angular velocities allong the vertical axis and the horizontal plane.
Description
95th percentile of the angular velocities of the feet and the arms the vertical axis and the horizontal plane.
Time Frame
Month 0 to month 24
Title
Change in the acceleration's entropy
Description
Acceleration's entropy computed in the different axis of the feet and the arms.
Time Frame
Month 0 to month 24

10. Eligibility

Sex
All
Maximum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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.)
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Isabelle DESGUERRE, MD, PhD
Phone
1 44 49 48 56
Ext
+33
Email
isabelle.desguerre@aphp.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Hélène MOREL
Phone
1 71 19 63 46
Ext
+33
Email
helene.morel@aphp.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Isabelle DESGUERRE, MD, PhD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Brian TERVIL, PhD
Organizational Affiliation
Centre Borelli - Université Paris Descartes
Official's Role
Study Director
Facility Information:
Facility Name
Hôpital Necker-Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Isabelle DESGUERRE, MD, PhD
Phone
1 44 49 48 56
Ext
+33
Email
isabelle.desguerre@aphp.fr
First Name & Middle Initial & Last Name & Degree
Rémi BARROIS, PhD
First Name & Middle Initial & Last Name & Degree
Christine BARNERIAS, MD

12. IPD Sharing Statement

Plan to Share IPD
No

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

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