UMIN-ICDS Clinical Trial

Public title

Psychosocial support program for patients with myotonic dystrophy and their caregivers: a randomized control trial

Acronym

Psychosocial support program for patients with myotonic dystrophy and their caregivers: a randomized control trial

Scientific Title

Psychosocial support program for patients with myotonic dystrophy and their caregivers: a randomized control trial

Scientific Title:Acronym

Psychosocial support program for patients with myotonic dystrophy and their caregivers: a randomized control trial

Region

Japan

Condition

myotonic dystrophy type 1

Classification by specialty

Neurology

Classification by malignancy

Others

Genomic information

YES

Narrative objectives1

Myotonic dystrophy type 1 (DM1) is a rare autosomal dominant disorder, which presents with a wide range of symptoms, including muscle weakness and myotonia, as well as cognitive dysfunction including apathy, pain, excessive daytime sleepiness, and fatigue. It is important not only to address physical symptoms but also to implement approaches that alleviate symptoms through psychological and cognitive interventions and promote activity and social participation. However, intervention studies on psychosocial care in muscular dystrophy are limited. In addition, caregivers often face considerable challenges in coping with the diverse symptoms experienced by patients. Nevertheless, research focusing on the care and support needs of caregivers themselves remains inadequate.
We developed the "Psychosocial Self-Care Program for Patients with DM1 and Their Caregivers" and conducted an exploratory intervention study (the pilot study) to examine its feasibility. This program aims to help participants understand the importance and methods of psychosocial self-care, thereby increasing patients' activity levels, alleviating disease-specific symptoms such as fatigue and excessive daytime sleepiness, and reducing caregiver burden. Results of the pilot study indicated that mean scores for patients' activity frequency and caregivers' burden improved following the intervention. On the other hand, fatigue scores worsened among participants who reported deterioration in motor function during participation in the program.
Based on these findings, we will conduct an efficacy study of a revised program, an increased number of participants and study sites, as well as the inclusion of a control group. To date, no published efficacy studies have examined psychosocial support programs for patients with muscular diseases in Japan. Therefore, conducting this study will contribute to expanding care options for patients with DM1 and their caregivers in Japan.

Basic objectives2

Efficacy

Basic objectives -Others

Trial characteristics_1

Trial characteristics_2

Developmental phase

Primary outcomes

The total score of the Frenchay Activities Index (FAI), a measure of the level of activity and participation. Assessments will be conducted at baseline, 10 months, and 13 months.

Key secondary outcomes

ADL / Muscular impairment
* Barthel Index (baseline)
* Muscular Impairment Rating Scale (MIRS) (baseline)

DM1-specific symptoms
* Fatigue Severity Scale (FSS) (baseline, 10 months, and 13 months)
* Epworth Sleepiness Scale (ESS) (baseline, 10 months, and 13 months)
* Apathy Evaluation Scale, caregiver version (AES) (baseline, 10 months, and 13 months)

QOL / Patients' perceptions of their illness
* SF-12v2 (baseline, 10 months, and 13 months)
* Individualized Neuromuscular Quality of Life Questionnaire (INQoL) (baseline, 10 months, and 13 months)

Cognitive function / Intellectual standard
* Japanese Adult Reading Test (JART25) (baseline)
* Mini-Mental State Examination (MMSE) (baseline, 10 months, and 13 months)

Caregiver burden
* Japanese version of the shortened Zarit Caregiver Burden Index (J-ZBI8) (baseline, 10 months, and 13 months)

Study type

Interventional

Basic design

Parallel

Randomization

Randomized

Randomization unit

Individual

Blinding

Open -no one is blinded

Control

No treatment

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

2

Purpose of intervention

Educational,Counseling,Training

Type of intervention

Behavior,custom

Other

Interventions/Control_1

[Intervention Group]
A psychosocial support program will be implemented. The program, entitled "Psychosocial Self-Care Program for Patients with Myotonic Dystrophy Type 1 and Their Caregivers" is designed to help patients and caregivers learn self-care strategies for promoting mental health and social participation together with healthcare professionals. The study will examine whether participation in the program leads to increased activity levels and improved quality of life (QOL) among patients. In addition, caregivers will learn stress-coping strategies and ways to seek support for themselves, and the study will assess whether participation reduces caregiver burden. The program will use specially developed text materials and worksheets and will be conducted in an interactive dialogue format between healthcare professionals and study participants. Sessions will be held once monthly (+/-14 days), for a maximum of 12 sessions, to be completed within 12 months from baseline.

Interventions/Control_2

[Control Group]
Participants will be provided with an informational booklet on general psychosocial health and self-care, which patients and caregivers will be asked to read.

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

18

years-old

<=

Age-upper limit

Not applicable

Gender

Male and Female

Key inclusion criteria

1. Patients and caregivers who are able to sufficiently understand the contents of the study information sheet and from whom written informed consent has been obtained prior to study initiation.
2. Both the patient and the caregiver have agreed to participate in the study.
3. Patients and caregivers aged 18 years or older at the time informed consent is obtained.
4. Patients diagnosed with DM1 based on genetic testing of the *DMPK* gene.
5. Patients who are able to move independently, either by walking or with the use of assistive devices and/or a wheelchair.
6. "Caregiver" refers to a family member, relative, partner, or friend who lives with the patient, or, in the case of patients with long-term hospitalization, the family member, relative, partner, or friend who is closest to the patient. The term includes any person who helps reduce the burden experienced by the patient due to the disease, regardless of the extent of caregiving provided.
7. The caregiver is aware that the patient has been diagnosed with DM1.

Key exclusion criteria

1. Either the patient or the caregiver does not wish to participate in the study.
2. Patient and/or caregiver is taking antipsychotic medication and/or mood stabilizers.
3. Patient and/or caregiver is taking antidepressants, anxiolytics, hypnotics/sleep medications, Modafinil, or Ritalin, and the dosage has changed within the past 12 months. However, as-needed use of anxiolytics and sleep medications will not be considered exclusion criteria.
4. Caregiver is a professional caregiver.
5. Cases judged by the principal investigator or sub-investigators to be unsuitable for participation in the study for any other reason.
6. Participant is enrolled in another interventional study, clinical trial, or observational study.

Target sample size

100

Name of lead principal investigator

1st name	Makiko
Middle name
Last name	Endo

Organization

National Center of Neurology and Psychiatry

Division name

NCNP Hospital, Department of Clinical Research Support

Zip code

187-8551

Address

4-1-1 Ogawahigashi-cho, Kodaira, Tokyo, Japan

TEL

042-341-2711

Email

endo@ncnp.go.jp

Name of contact person

1st name	Makiko
Middle name
Last name	Endo

Organization

National Center of Neurology and Psychiatry

Division name

NCNP Hospital, Department of Clinical Research Support

Zip code

187-8551

Address

4-1-1 Ogawahigashi-cho, Kodaira, Tokyo, Japan

TEL

042-341-2711

Homepage URL

Email

endo@ncnp.go.jp

Institute

National Center of Neurology and Psychiatry

Institute

Department

Personal name

Organization

JSPS Kakenhi

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Ethics Committee, National Center of Neurology and Psychiatry

Address

4-1-1 Ogawahigashi-cho, Kodaira, Tokyo

Tel

042-341-2711

Email

https://www.ncnp.go.jp/research-info/

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

国立精神・神経医療研究センター病院（東京都）
国立病院機構　青森病院（青森県）　
大阪大学医学部附属病院／大阪大学大学院（大阪府）
国立病院機構　大阪刀根山医療センター（大阪府）
国立病院機構　鈴鹿病院（三重県）
国立病院機構　仙台西多賀病院（宮城県）

Date of disclosure of the study information

2026

Year

05

Month

22

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

Preinitiation

Date of protocol fixation

2026

Year

05

Month

12

Day

Date of IRB

2026

Year

05

Month

12

Day

Anticipated trial start date

2026

Year

08

Month

01

Day

Last follow-up date

2029

Year

03

Month

31

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2026

Year

05

Month

22

Day

Last modified on

2026

Year

05

Month

22

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000070551