UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000033935 |
|---|---|
| Receipt number | R000038620 |
| Scientific Title | Explore evaluation methods of Nusinersen efficacy on advanced SMA patients with limited gross motor function |
| Date of disclosure of the study information | 2018/08/29 |
| Last modified on | 2022/03/02 09:33:49 |
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Basic information
Public title
Explore evaluation methods of Nusinersen efficacy on advanced SMA patients with limited gross motor function
Acronym
NEOSMA Study
Scientific Title
Explore evaluation methods of Nusinersen efficacy on advanced SMA patients with limited gross motor function
Scientific Title:Acronym
NEOSMA Study
Region
| Japan |
Condition
Condition
Spinal Muscular Atrophy(SMA)
Classification by specialty
| Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To investigate whether cognitive ability measured by the matching-pair test through eye movements is improved by Nusinersen treatment in advanced SMA patients with significantly limited gross motor function
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Matching pair test:
Changes in scores (24 weeks after baseline)
Changes in time taken for matching pair test (24 weeks after baseline)
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
| 3 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1.Type-1 SMA patients who can provide written consent for participation in the study, whose ages are 3 years and older
2.Type-1 SMA patients who receive Nusinersen treatment or are determined to be subject to treatment
3.Type-1 SMA patients who are naive, and non-ambulatory, and Hammersmith Functional Motor Scale Expanded (HFMSE) score = 0 ,before receiving treatment
Key exclusion criteria
1.Past medical history of Hypoxic brain injury or epileptic episode
2.Present respiratory infection
3.Patients who cannot perform matching test by eye gaze apparatus: unable to open eyes and to fix the eye gaze for the system
Target sample size
10
Research contact person
Name of lead principal investigator
| 1st name | Kotaro |
| Middle name | |
| Last name | Yuge |
Organization
Kurume University School of Medicine
Division name
Department of Pediatrics
Zip code
830-0011
Address
67 Asahi-machi, Kurume city, Fukuoka
TEL
0942-35-3311
yugekotaro@med.kurume-u.ac.jp
Public contact
Name of contact person
| 1st name | Satoru |
| Middle name | |
| Last name | Enomoto |
Organization
Mebix, Inc.
Division name
Research Promotion Group
Zip code
107-0052
Address
Akasaka Intercity,1-11-44 Akasaka, Minato-ku,Tokyo
TEL
03-4362-4504
Homepage URL
neosma@mebix.co.jp
Sponsor or person
Institute
Biogen Japan Ltd.
Kurume University School of Medicine
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
Kurume University Ethic Committee
Address
67 Asahi-machi, Kurume city, Fukuoka
Tel
0942-31-7917
i_rinri@kurume-u.ac.jp
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
Other administrative information
Date of disclosure of the study information
| 2018 | Year | 08 | Month | 29 | 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
No longer recruiting
Date of protocol fixation
| 2018 | Year | 07 | Month | 06 | Day |
Date of IRB
| 2018 | Year | 08 | Month | 20 | Day |
Anticipated trial start date
| 2018 | Year | 10 | Month | 29 | Day |
Last follow-up date
| 2020 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Nusinersen, the first therapeutic agent for patients with spinal muscular atrophy (SMA), has been available in Japan just as in other countries, and has been given to many SMA patients so far. After being treated with Nusinersen, families and attending physicians observed some favorable improvements in the symptoms of patients, including respiratory activity, sleeping status, and communication via eye movement. However, these improvements in symptoms have not been adequately evaluated by currently used motor function tests, such as the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score, to assess the efficacy of Nusinersen. Therefore, the development of a new method to appropriately evaluate the efficacy of Nusinersen is a pressing need for SMA patients.
In this research, we will examine whether the effects of Nusinersen treatment (other than motor function) can be determined by using a test called a matching pair test.
Management information
Registered date
| 2018 | Year | 08 | Month | 29 | Day |
Last modified on
| 2022 | Year | 03 | Month | 02 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038620
| Research Plan | |
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| Registered date | File name |
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| Registered date | File name |
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