usinersen Effect on Ambulatory Function of Patients with Late-onset, Chronic SMA: A Multicenter, Prospective and Retrospective Observational Study.

Not Applicable

Completed

• Conditions: SMA patients with limited ambulation, a minimum of 40 months disease duration, and >=100cm in height

• Registration Number: JPRN-jRCT1090220400
• Lead Sponsor: Takashi Nakajima

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-12-07
• Study Completion: 2021-01-25
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

To be eligible to participate in this study, candidates must meet the following eligibility criteria.
Patients before or during treatment with nusinersen in daily clinical practice.
Able to provide consent document. For patients aged 16 or older and under 20, informed consent by legal guardian is required in addition to him/her consent. For patients under 16, obtaining informed assent by him/her is encouraged after explaining the study in readily comprehensible language.
Patients with later-onset, genetically confirmed 5q SMA, having a minimum disease duration of 40 months at the time of treatment initiation.
Able to stand with assistance but unable to walk independently without support.
>=100 cm in height at the time of treatment initiation.
Naive to physical therapy using HAL at screening (except for a HAL monotherapy cohort).

Exclusion Criteria

Patients with comorbid diseases including dyspnea on exertion,heart failure, arrhythmia, or myocardial infarction.
Patients physically unfit to wearing HAL or having difficulty in using HAL due to severe deformation of the body (for the group using HAL).
Female who are pregnant or breastfeeding at screening or who wish to be pregnant and unwilling to use effective contraceptive measures for the duration of the study.
Patients judged to be ineligible to the study at the discretion of the principal physician investigator or the physician sub-investigators.

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
2-minute walk test

Secondary Outcome Measures

Name	Time	Method
6-minute walk test<br>10-meter walk test<br>Hammersmith Functional Motor Scale-Expanded<br>Functional Independence Measure<br>Revised Upper Limb Module<br>PedsQL<br>Decision Regret Scale