Holter of Movement in Patients With SMA Undergoing Treatment.

Spinal Muscular Atrophy Clinical Trial

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.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04888702
• Other study ID #: ActiSMA.
• Status: Active, not recruiting
• Phase: N/A
• Start date: August 8, 2018
• Est. completion date: July 30, 2024

Study information

• Verified date: May 2024
• Source: Centre Hospitalier Universitaire de Liege
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: July 30, 2024
• Est. primary completion date: July 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

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.

Locations

Country	Name	City	State
Belgium	Centre Hospitalier Régional de la Citadelle	Liège

Sponsors (5)

Lead Sponsor	Collaborator
Centre Hospitalier Universitaire de Liege	Centre Hospitalier Régional de la Citadelle, Erasme University Hospital, Queen Fabiola Children's University Hospital, SYSNAV

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	95th centile of stride length	95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).	39 months
Primary	50th centile of stride length	50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).	39 months
Primary	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	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
Secondary	Dynamometric measures	Assessment of upper limb strength with myogrip and myopinch (kg).	39 months
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Hamersmith funcitonnal motor scale (HFMS)	HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy.	39 months
Secondary	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