Holter of Movement in Patients with SMA Undergoing Treatment.

Holter of Movement in Patients with Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment with Spinraza ° or Risdiplam. Acti-SMA Study.

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Spinal Muscular Atrophy
• Intervention / Treatment: Device: Actimyo

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Belgium
  • Bruxelles, Belgium, 1000
    • Erasme Hospital - Cliniques universitaires de Bruxelles
  • Liège, Belgium, 4000
    • Centre Hospitalier Régional de la Citadelle
• Romania
  • Bucharest, Romania
    • National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu"

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SMA patients; Patients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment.	Device: Actimyo; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
95th centile of stride velocity	95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life. (meter per second).	39 months
50th centile of stride velocity	50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).	39 months
95th centile of stride length	95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).	39 months
50th centile of stride length	50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).	39 months
Upper limb function evolution in patient with SMA undergoing treatment	Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.	39 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	39 months
Dynamometric measures	Assessment of upper limb strength with myogrip and myopinch (kg).	39 months
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).	39 months
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).	39 months
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).	39 months
revised upper limb module (RULM)	Revised Upper Limb Module (RULM) is an assessment specifically designed for upper limb function in Spinal Muscular Atrophy.	39 months
Hamersmith funcitonnal motor scale (HFMS)	HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy.	39 months
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.	39 months

Collaborators and Investigators

• Sponsor: Centre Hospitalier Universitaire de Liege
• Collaborators: Queen Fabiola Children's University Hospital; Erasme University Hospital; Centre Hospitalier Régional de la Citadelle; SYSNAV
• Investigators: Principal Investigator: Margaux Poleur, CHR Citadelle-CHU liège

Study record dates

Study Major Dates

• Study Start (Actual): August 8, 2018
• Primary Completion (Actual): January 30, 2025
• Study Completion (Estimated): May 30, 2025

Study Registration Dates

• First Submitted: March 11, 2021
• First Submitted That Met QC Criteria: May 11, 2021
• First Posted (Actual): May 17, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 10, 2025
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Keywords: Accelerometry; Nusinersen; Actimyo; Home monitoring; Motor neuron disease; Risdiplam
• Additional Relevant MeSH Terms: Neurologic Manifestations; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; Motor Neuron Disease; Atrophy; Muscular Atrophy; Muscular Atrophy, Spinal
• Other Study ID Numbers: ActiSMA.

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No