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Holter of Movement in Patients With SMA Undergoing Treatment.

Primary Purpose

Spinal Muscular Atrophy

Status
Active
Phase
Not Applicable
Locations
Belgium
Study Type
Interventional
Intervention
Actimyo
Sponsored by
Dr. Stéphanie Delstanche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Spinal Muscular Atrophy focused on measuring Actimyo, Accelerometry, Motor neuron disease, Risdiplam, Nusinersen, Home monitoring

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Genetically confirmed spinal muscular atrophy.
  • Treatment by Spinraza ° or by risdiplam planned.
  • Over 6 years old.
  • Signed informed consent.

Exclusion Criteria:

- Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.

Sites / Locations

  • Centre Hospitalier Régional de la Citadelle

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

SMA patients

Arm Description

Patients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment.

Outcomes

Primary Outcome Measures

95th centile of stride velocity
95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life. (meter per second).
50th centile of stride velocity
50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
95th centile of stride length
95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
50th centile of stride length
50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Upper limb function evolution in patient with SMA undergoing treatment
Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.

Secondary Outcome Measures

6-minute walk test
The Six-Minute Walk Test is a functional walking test in which investigators assess the distance the patient can walk in six minutes (meter).
Dynamometric measures
Assessment of upper limb strength with myogrip and myopinch (kg).
10-metre walk test
The 10-metre walk test is a functional walking test in which investigators assess the time the patient take to walk 10 meters (second).
rise from floor
The rise from floor is a functional test in which investigators assess the time the patient take to rise from the floor (second).
4-stair climb
The 4-stair climbr is a functional test in which investigators assess the time the patient take to climb a four-step staircase (second).
revised upper limb module (RULM)
Revised Upper Limb Module (RULM) is an assessment specifically designed for upper limb function in Spinal Muscular Atrophy.
Hamersmith funcitonnal motor scale (HFMS)
HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy.
motor function measure (MFM)
Motor Function Measure is a global quantitative scale created to measure the functional motor abilities of a person affected by a neuromuscular disease.

Full Information

First Posted
March 11, 2021
Last Updated
October 6, 2023
Sponsor
Dr. Stéphanie Delstanche
Collaborators
Sysnav, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Universitaire de Liege
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1. Study Identification

Unique Protocol Identification Number
NCT04888702
Brief Title
Holter of Movement in Patients With SMA Undergoing Treatment.
Official Title
Holter of Movement in Patients With Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment With Spinraza ° or Risdiplam. Acti-SMA Study.
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 8, 2018 (Actual)
Primary Completion Date
April 30, 2024 (Anticipated)
Study Completion Date
April 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Dr. Stéphanie Delstanche
Collaborators
Sysnav, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Universitaire de Liege

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
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. A secondary objective would also be to identify suitable accelerometric measurements that are sensitive to change but also well correlated to other clinical scores.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy
Keywords
Actimyo, Accelerometry, Motor neuron disease, Risdiplam, Nusinersen, Home monitoring

7. Study Design

Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
all patients undergo clinical evaluation and wear Actimyo device in daily living to evaluate their improvement under treatment.
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SMA patients
Arm Type
Experimental
Arm Description
Patients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment.
Intervention Type
Device
Intervention Name(s)
Actimyo
Intervention Description
Actimyo° is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle.
Primary Outcome Measure Information:
Title
95th centile of stride velocity
Description
95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life. (meter per second).
Time Frame
39 months
Title
50th centile of stride velocity
Description
50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
Time Frame
39 months
Title
95th centile of stride length
Description
95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Time Frame
39 months
Title
50th centile of stride length
Description
50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Time Frame
39 months
Title
Upper limb function evolution in patient with SMA undergoing treatment
Description
Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.
Time Frame
39 months
Secondary Outcome Measure Information:
Title
6-minute walk test
Description
The Six-Minute Walk Test is a functional walking test in which investigators assess the distance the patient can walk in six minutes (meter).
Time Frame
39 months
Title
Dynamometric measures
Description
Assessment of upper limb strength with myogrip and myopinch (kg).
Time Frame
39 months
Title
10-metre walk test
Description
The 10-metre walk test is a functional walking test in which investigators assess the time the patient take to walk 10 meters (second).
Time Frame
39 months
Title
rise from floor
Description
The rise from floor is a functional test in which investigators assess the time the patient take to rise from the floor (second).
Time Frame
39 months
Title
4-stair climb
Description
The 4-stair climbr is a functional test in which investigators assess the time the patient take to climb a four-step staircase (second).
Time Frame
39 months
Title
revised upper limb module (RULM)
Description
Revised Upper Limb Module (RULM) is an assessment specifically designed for upper limb function in Spinal Muscular Atrophy.
Time Frame
39 months
Title
Hamersmith funcitonnal motor scale (HFMS)
Description
HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy.
Time Frame
39 months
Title
motor function measure (MFM)
Description
Motor Function Measure is a global quantitative scale created to measure the functional motor abilities of a person affected by a neuromuscular disease.
Time Frame
39 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Genetically confirmed spinal muscular atrophy. Treatment by Spinraza ° or by risdiplam planned. Over 6 years old. Signed informed consent. Exclusion Criteria: - Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Margaux Poleur
Organizational Affiliation
CHR Citadelle-CHU liège
Official's Role
Principal Investigator
Facility Information:
Facility Name
Centre Hospitalier Régional de la Citadelle
City
Liège
ZIP/Postal Code
4000
Country
Belgium

12. IPD Sharing Statement

Plan to Share IPD
No

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Holter of Movement in Patients With SMA Undergoing Treatment.

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