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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000033935
Receipt No. 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 2018/11/07

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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
Middle name
Last name Kotaro Yuge
Organization Kurume University School of Medicine
Division name Department of Pediatrics
Zip code
Address 67 Asahi-machi, Kurume city, Fukuoka
TEL 0942-35-3311
Email yugekotaro@med.kurume-u.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Yuki Nishiura
Organization Mebix, Inc.
Division name Research Promotion Group
Zip code
Address Akasaka Intercity,1-11-44 Akasaka, Minato-ku,Tokyo
TEL 03-4362-4504
Homepage URL
Email neosma@mebix.co.jp

Sponsor
Institute Biogen Japan Ltd.

Kurume University School of Medicine
Institute
Department

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
Address
Tel
Email

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 Open public recruiting
Date of protocol fixation
2018 Year 07 Month 06 Day
Date of IRB
Anticipated trial start date
2018 Year 10 Month 29 Day
Last follow-up date
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
2018 Year 11 Month 07 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038620

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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