UMIN-CTR Clinical Trial

Public title

Study about biomarkers of female carriers in spinal and bulbar muscular atrophy(SBMA)

Acronym

Biomarkers of female carriers in SBMA

Scientific Title

Study about biomarkers of female carriers in spinal and bulbar muscular atrophy(SBMA)

Scientific Title:Acronym

Biomarkers of female carriers in SBMA

Region

Japan

Condition

Spinal and bulbar muscular atrophy

Classification by specialty

Neurology

Adult

Classification by malignancy

Others

Genomic information

YES

Narrative objectives1

In this study, we aim to evaluate the biomarkers in female carriers of spinal and bulbar muscular atrophy (SBMA) and compare them with those of healthy female controls.

Basic objectives2

Others

Basic objectives -Others

To explore and identify the biomarkers of female carriers in spinal and bulbar muscular atrophy (SBMA)

Trial characteristics_1

Exploratory

Trial characteristics_2

Others

Developmental phase

Not applicable

Primary outcomes

Composite measure that combines quantitative muscle strength measurements

Key secondary outcomes

(1) Motor function: Grip power, tongue pressure, Timed walk test, Rise from bed test, and modified QMG Score, Manual muscle test
(2) Respiratory function test: vital capacity, forced vital capacity, forced expiratory volume one second percent, peak expiratory flow and V50/V25
(3)Motor unit number estimation (MUNE) by stimulating the ulnar nerve
(4) Activities of daily living(ADL) assessed by ALSFRS-R and SBMAFRS questionnaires
(5) Swallowing Disturbance Questionnaire
(6) Quality of life questionnaires: SWAL-QOL and ALSAQ-5
(7) Multidimensional Fatigue Inventory
(8) Muscle mass measured by dual-energy X-ray absorptiometry
(9) Urinary 8-OHdG
(10)Electromyography
(11)Electrocardiogram

Study type

Observational

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

Age-lower limit

20

years-old

<=

Age-upper limit

Not applicable

Gender

Female

Key inclusion criteria

1.Female carriers of SBMA
a) Mothers or daughters of patients who were diagnosed as SBMA by a genetic test
b) Females who are more than 20 years old at the time of the agreement acquisition
c) As a principle, females who are married and who have no children who are under the age of 20
d) Females who understand that they are carriers of SBMA
e) Females with written informed consent
2.Healthy female controls
a) Females who are 20 years of age or older at the time of the agreement acquisition
b) Females who aren't affected with neuromuscular diseases and other severe diseases
c) Females with written informed consent

Key exclusion criteria

a) Females who have desire to mother a child.
b) Females who have severe complications
c) Females who are considered as being not appropriate to participate in the study

Target sample size

40

Name of lead principal investigator

1st name
Middle name
Last name	Masahisa Katsuno

Organization

Nagoya University Graduate School of Medicine

Division name

Department of Neurology

Zip code

Address

65 Tsurumai-cho, Showa-ku, Nagoya 466-8550 Japan

TEL

052-744-2385

Email

Ka2no@med.nagoya-u.ac.jp

Name of contact person

1st name
Middle name
Last name	Tomonori Inagaki

Organization

Nagoya University Graduate School of Medicine

Division name

Department of Neurology

Zip code

Address

65 Tsurumai-cho, Showa-ku, Nagoya 466-8550 Japan

TEL

052-744-2390

Homepage URL

Email

t.inagaki@med.nagoya-u.ac.jp

Institute

Department of Neurology, Nagoya University Graduate School of Medicine

Institute

Department

Personal name

Organization

Japan Society for the Promotion of Science

Organization

Division

Category of Funding Organization

Other

Nationality of Funding Organization

Co-sponsor

Jichi medical university

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

名古屋大学医学部附属病院(愛知県)、自治医科大学（栃木県）、

Date of disclosure of the study information

2016

Year

01

Month

26

Day

URL releasing protocol

Publication of results

Unpublished

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

Recruitment status

Open public recruiting

Date of protocol fixation

2015

Year

11

Month

10

Day

Date of IRB

Anticipated trial start date

2016

Year

02

Month

01

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Study design: case-control study
Objective: The carriers and healty controls who have written-informed consent in Nagoya University Hospital and Jichi medical university between 2016/2/1 and 2020/3/31.
Measurements: blood, urinary, clinical data

Registered date

2016

Year

01

Month

25

Day

Last modified on

2018

Year

02

Month

19

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023437