SMA patients with limited ambulation, a minimum of 40 months disease duration, and >=100cm in height
Conditions
Interventions
Intervention type:
Name of intervention:
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Sponsors
Takashi Nakajima
Toshio Saito,Akihiro Hashiguchi
Eligibility
Sex/Gender
All
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 2-minute walk test | — |
Secondary
| Measure | Time frame |
|---|---|
| 6-minute walk test 10-meter walk test Hammersmith Functional Motor Scale-Expanded Functional Independence Measure Revised Upper Limb Module PedsQL Decision Regret Scale | — |
Countries
Japan
Contacts
Public ContactTakashi Nakajima
Niigata National Hospital, National Hospital Organization
Outcome results
None listed