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

Email

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


Email

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

Email

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


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name