UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000042015 |
|---|---|
| Receipt number | R000047950 |
| Scientific Title | Japan REgistry for Adult subjeCTs of SMA |
| Date of disclosure of the study information | 2020/10/07 |
| Last modified on | 2023/04/13 12:03:03 |
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Basic information
Public title
Japan REgistry for Adult subjeCTs of SMA
Acronym
jREACT-SMA
Scientific Title
Japan REgistry for Adult subjeCTs of SMA
Scientific Title:Acronym
jREACT-SMA
Region
| Japan |
Condition
Condition
Spinal Muscular Atrophy
Classification by specialty
| Neurology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To set up a registry to collect longitudinal data on 5q-SMA adult patients in Japan on both the natural history of the disease and the outcome of patients under treatment
Basic objectives2
Others
Basic objectives -Others
To set up a registry
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Collecting longitudinal data of Motor function and QOL/PRO
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
1)Patients with clinically diagnosed SMA, aged 18 years and older at the time of enrollment.
2)SMA patients with genetically confirmed loss of the SMN1 gene as well as retention of at least one copy of the SMN2 gene.
3)SMA patients who are available for follow-up visits.
4)SMA patients who can provide written consent for participation in the study.
Key exclusion criteria
1)SMA patients who have psychiatric disorder.
2)SMA patients who are judged as unsuitable for the study by the investigator.
Target sample size
150
Research contact person
Name of lead principal investigator
| 1st name | Masahisa |
| Middle name | |
| Last name | Katsuno |
Organization
Nagoya University Graduate School of Medicine
Division name
Department of Neurology
Zip code
466-8550
Address
65 Tsurumai Showa, Nagoya, Aichi, Japan
TEL
052-744-2389
ka2no@med.nagoya-u.ac.jp
Public contact
Name of contact person
| 1st name | Satona |
| Middle name | |
| Last name | Otake |
Organization
Mebix, Inc.
Division name
Research Promotion Group
Zip code
107-0052
Address
Akasaka Intercity,1-11-44 Akasaka, Minato-ku,Tokyo, Japan
TEL
03-4362-4500
Homepage URL
sma_adult_registry@mebix.co.jp
Sponsor or person
Institute
Nagoya University Graduate School of Medicine
Biogen Japan Ltd.
Chugai Pharmaceutical Co., Ltd.
Institute
Department
Personal name
Funding Source
Organization
Biogen Japan Ltd.
Chugai Pharmaceutical Co., 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
The Ethical Review Committee of Nagoya University
Address
65 Tsurumai Showa, Nagoya Aichi
Tel
052-744-2479
iga-shinsa@adm.nagoya-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
| 2020 | Year | 10 | Month | 07 | 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
| 2020 | Year | 09 | Month | 18 | Day |
Date of IRB
| 2020 | Year | 09 | Month | 28 | Day |
Anticipated trial start date
| 2020 | Year | 11 | Month | 01 | Day |
Last follow-up date
| 2025 | Year | 11 | Month | 30 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
SMA therapy with nusinersen and risdiplam has been approved principally based on the results of clinical studies in SMA infants and children. Besides its potential therapeutic utility for all types of SMA, the drug could in principle also be beneficial for the chronic progressors who grow into adulthood. To our knowledge, this medicinal capacity for the adult patients has not been fully evaluated. Furthermore, there has been no comprehensive, prospective analysis of the clinical course of SMA at adult stages. A multidisciplinary assessment of SMA adults will allow us to address the current gaps in our knowledge of SMA natural history during adult life as well as the drug availability for patients from prodromal and early to advanced adult stages. Establishing a multicenter registry of SMA adults throughout Japan, we will take the lead in organizing cross-sectional and prospective longitudinal analyses of SMA. This study is intended to aid in a better understanding of the pathophysiology of SMA and provide clear indications of SMA therapy at adult stages, thereby overcoming some of the potential therapeutic limitations.
Management information
Registered date
| 2020 | Year | 10 | Month | 05 | Day |
Last modified on
| 2023 | Year | 04 | Month | 13 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000047950
