UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000061891 |
|---|---|
| Receipt number | R000069804 |
| Scientific Title | A multicenter observational study for the higher dose regimen of Nusinersen |
| Date of disclosure of the study information | 2026/06/12 |
| Last modified on | 2026/06/12 16:42:17 |
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Basic information
Public title
A multicenter observational study for the higher dose regimen of Nusinersen
Acronym
A multicenter observational study for the higher dose regimen of Nusinersen
Scientific Title
A multicenter observational study for the higher dose regimen of Nusinersen
Scientific Title:Acronym
A multicenter observational study for the higher dose regimen of Nusinersen
Region
| Japan |
Condition
Condition
Participants with later-onset 5q spinal muscular atrophy (SMA)
Classification by specialty
| Neurology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To evaluate the therapeutic effects of higher dose nusinersen on patient-reported health status and physical function in individuals with later-onset SMA
Basic objectives2
Others
Basic objectives -Others
Observational study
Trial characteristics_1
Others
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
Change from baseline to 12 months in MFI-20 total score
Key secondary outcomes
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| 18 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Patients must meet all of the following criteria:
(1) Patients who are scheduled to initiate or have already initiated high-dose nusinersen under routine clinical practice.
(2) Patients who are able to provide written informed consent by themselves.
Patients must be able to understand the purpose and risks of the study, and written informed consent must be obtained in accordance with national and local data protection laws.
If the patient has sufficient decision-making capacity but has difficulty writing due to progression of the underlying disease, written informed consent may be obtained by proxy writing, provided that the patient's willingness to participate is confirmed and a witness is present.
(3) Patients with a genetically confirmed diagnosis of late-onset 5q spinal muscular atrophy (SMA) (Type II, Type III, or Type IV).
(4) Patients who are 18 years of age or older at the time of obtaining informed consent.
(5) Patients who are able to attend follow-up visits at least once per year during the study period.
Key exclusion criteria
Candidates will be excluded from study entry if any of the following criteria apply at screening:
(1) Medical or psychiatric conditions that may interfere with the ability to comply with study procedures or assessments.
(2) Current enrollment in ASCEND or any other investigational nusinersen trial.
(3) Current or planned enrollment in an investigational trial for SMN-directed therapy or SMA gene therapy.
(4) Patients who are deemed inappropriate as observational study participants by the principal investigator or a sub-investigator.
Target sample size
40
Research contact person
Name of lead principal investigator
| 1st name | Hiroshi |
| Middle name | |
| Last name | Takashima |
Organization
Kagoshima University Graduate School of Medical and Dental Sciences
Division name
Department of Neurology and Geriatrics
Zip code
099-275-5111
Address
8-35-1 Sakuragaoka, Kagoshima-shi, Kagoshima
TEL
099-275-5111
thiroshi@m3.kufm.kagoshima-u.ac.jp
Public contact
Name of contact person
| 1st name | Rie |
| Middle name | |
| Last name | Yokokawa |
Organization
EPS Corporation
Division name
Clinical Research Center, RWE Division
Zip code
564-0063
Address
4th Floor, Maruito Daini Esaka Building 1-17-6 Esaka-cho, Suita-shi, Osaka
TEL
06-7176-5731
Homepage URL
prj-hd-nus-office@eps.co.jp
Sponsor or person
Institute
Kagoshima University Graduate School of Medical and Dental Sciences
Institute
Department
Personal name
Funding Source
Organization
Biogen Japan Ltd.
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Non-Profit Organization MINS Institutional Review Board
Address
2F, 1-15-14 Dogenzaka, Shibuya-ku, Tokyo 150-0043, Japan
Tel
03-6416-1868
npo-mins@j-irb.com
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
鹿児島大学病院（鹿児島県）、名古屋大学医学部附属病院（愛知県）、国立病院機構新潟病院（新潟県）、大阪刀根山医療センター（大阪府）、東京大学医学部附属病院（東京都）、横浜市立大学附属病院（神奈川県）
Other administrative information
Date of disclosure of the study information
| 2026 | Year | 06 | Month | 12 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Open public recruiting
Date of protocol fixation
| 2026 | Year | 02 | Month | 25 | Day |
Date of IRB
| 2026 | Year | 03 | Month | 19 | Day |
Anticipated trial start date
| 2026 | Year | 06 | Month | 12 | Day |
Last follow-up date
| 2027 | Year | 12 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by abnormalities in the SMN1 gene, leading to progressive muscle weakness and atrophy. Nusinersen is an antisense oligonucleotide therapy that increases the production of SMN protein derived from the SMN2 gene. The standard dose (12 mg) was approved in Japan in 2017 and has been widely used in patients with SMA.
In recent years, a higher-dose regimen of nusinersen (50/28 mg) demonstrated favorable tolerability and improvements in motor function in the international DEVOTE trial and was approved in Japan in September 2025. However, the DEVOTE trial was not designed to directly compare the higher-dose regimen with the standard dose, and evidence from real-world clinical practice remains limited, particularly for adult patients, severe cases, and non-ambulatory patients who were not sufficiently evaluated in clinical trials. In addition, there is an evidence gap regarding the switch from the Japan-specific standard regimen of 12 mg administered at 6-month intervals to the higher-dose regimen administered at 4-month intervals.
This study aims to generate post-marketing evidence by evaluating the effectiveness of higher-dose nusinersen in individuals with later-onset SMA under real-world clinical practice, using patient-reported outcomes and clinical assessments, thereby contributing to informed treatment selection and optimization of dosing regimens.
Management information
Registered date
| 2026 | Year | 06 | Month | 12 | Day |
Last modified on
| 2026 | Year | 06 | Month | 12 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000069804
