Brief Summary

There is no complete cure for SMA yet. However, the discovery of the genetic cause of SMA has led to the development of several treatment options that affect the genes involved in SMA - a gene replacement therapy called Zolgensma, and two drugs, called Nusinersen (Spinraza) and Risdiplam (Evyrsdi). In this context, the evaluation of efficacy and the long term follow-up of patients treated with these innovative treatments in clinical routine is one of the critical points. These evaluations are carried out in a medical context (clinical sites or research unit) using validated measurement tools and outcome measures. Carrying out these evaluations in a controlled environment can be considered from certain aspects as an advantage (reproducibility of measures, neutral environment, etc.), but also raises a certain number of questions regarding the impact on patients, the financial cost, or the relevance of the data obtained in an unnatural environment (stress, fatigue, patient motivation…). Also the regulatory authorities ask for longitudinal data for deciding to reimburse these expensive treatments. As such, the hospital cannot digest all these evaluations due to a lack of resources.

Trial Health

At Risk

Trial Health Score

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

Trial has exceeded expected completion date

Enrollment

participants targeted

Target at P25-P50 for not_applicable

Timeline

Completed

Started Dec 2023

Shorter than P25 for not_applicable

Geographic Reach

1 country

6 active sites

Status

unknown

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

2 months study duration

First Submitted

Initial submission to the registry

December 2, 2022

Completed

5 months until next milestone

First Posted

Study publicly available on registry

May 3, 2023

Completed

8 months until next milestone

Study Start

First participant enrolled

December 15, 2023

Completed

2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2024

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2024

Completed

Last Updated

December 1, 2023

Status Verified

November 1, 2023

Enrollment Period

2 months

First QC Date

December 2, 2022

Last Update Submit

November 30, 2023

Conditions

Muscular Atrophy, Spinal, Type II Muscular Atrophy, Spinal, Type III

Keywords

Muscular Atrophy, SpinalHome monitoringBioImpedance

Outcome Measures

Primary Outcomes (1)

To compare the results of physical evaluations between home and hospital
Evaluation of the correlations between results obtained during the physical evaluations at home compared to those obtained at hospital
Through study completion, an average of 3 weeks

Secondary Outcomes (8)

To determine the barriers for evaluation at home
Through study completion, an average of 3 weeks
Correlation of home and hospital muscle volume measurement methods
Through study completion, an average of 3 weeks
Correlation of home and hospital MyoGrip measurement
Through study completion, an average of 3 weeks
Correlation of home and hospital MyoPinch measurement
Through study completion, an average of 3 weeks
Correlation of home and hospital MFM results
Through study completion, an average of 3 weeks
+3 more secondary outcomes

Study Arms (2)

Home to onsite monitoring

OTHER

Patients will be monitored and evaluate in a first time at home then onsite.

Other: Strength force measurmentOther: Time testsOther: Motor scalesOther: QuestionnairesDevice: AccelerometryOther: MNROther: Bio-impedance analysis

Onsite to Home monitoring

OTHER

Patients will be monitored and evaluate in a first time onsite then at home .

Other: Strength force measurmentOther: Time testsOther: Motor scalesOther: QuestionnairesDevice: AccelerometryOther: MNROther: Bio-impedance analysis

Interventions

Strength force measurmentOTHER

The grip and pinch strength of the patient will be evaluate using dedicated devices (MyoGrip and MyoPinch)

Also known as: MyoGrip/Pinch