Tokyo, May 23 -- UMIN Clinical Trials Registry (UMIN-CTR) received information related to the study (UMIN000061661) titled 'Psychosocial support program for patients with myotonic dystrophy and their caregivers: a randomized control trial' on May 22.
Study Type:
Interventional
Study Design:
Basic Design - Parallel
Randomization - Randomized
Blinding - Open -no one is blinded
Control - No treatment
Primary Sponsor:
Institute - National Center of Neurology and Psychiatry
Condition:
Condition - myotonic dystrophy type 1
Classification by malignancy - Others
Genomic information - YES
Objective:
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
Intervention:
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.
Eligibility:
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 Size - 100
Recruitment Status:
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
To know more, visit https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000070551
Disclaimer: Curated by HT Syndication.
