NCT04888702

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.

Trial Health

58
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Aug 2018

Longer than P75 for not_applicable

Geographic Reach
2 countries

3 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 8, 2018

Completed
2.6 years until next milestone

First Submitted

Initial submission to the registry

March 11, 2021

Completed
2 months until next milestone

First Posted

Study publicly available on registry

May 17, 2021

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 30, 2025

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 30, 2025

Completed
Last Updated

February 12, 2025

Status Verified

May 1, 2024

Enrollment Period

6.5 years

First QC Date

March 11, 2021

Last Update Submit

February 10, 2025

Conditions

Spinal Muscular Atrophy

Keywords

ActimyoAccelerometryMotor neuron diseaseRisdiplamNusinersenHome monitoring

Outcome Measures

Primary Outcomes (5)

  • 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 Outcomes (8)

  • 6-minute walk test

    39 months

  • Dynamometric measures

    39 months

  • 10-metre walk test

    39 months

  • rise from floor

    39 months

  • 4-stair climb

    39 months

  • +3 more secondary outcomes

Study Arms (1)

SMA patients

EXPERIMENTAL

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

Device: Actimyo

Interventions

ActimyoDEVICE

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.

SMA patients

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Centre Hospitalier Régional de la Citadelle

Liège, Liège, 4000, Belgium

Location

Erasme Hospital - Cliniques universitaires de Bruxelles

Brussels, 1000, Belgium

Location

National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu"

Bucharest, Romania

Location

MeSH Terms

Conditions

Muscular Atrophy, SpinalMotor Neuron Disease

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Margaux Poleur

    CHR Citadelle-CHU liège

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Model Details: all patients undergo clinical evaluation and wear Actimyo device in daily living to evaluate their improvement under treatment.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Doctor

Study Record Dates

First Submitted

March 11, 2021

First Posted

May 17, 2021

Study Start

August 8, 2018

Primary Completion

January 30, 2025

Study Completion

May 30, 2025

Last Updated

February 12, 2025

Record last verified: 2024-05

Data Sharing

IPD Sharing
Will not share

Locations

BE(2)RO(1)