Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
A legfontosabb adatok
- Ki végzi
- Hanns Lochmuller
- Hol
- Kanada1 helyszín, 1 országbanVálassz országot a helyszínekhez
- Azonosító
- Állapot
- Fut, nem toboroz
- Résztvevők
- 13 fő, tervezett
- Kezdés
- 2025. márc. 25.
- Befejezés
- 2026. okt.
- A vizsgálat típusa
- Beavatkozással járó
- A vizsgált kezelések
- Music and movement education
- A regiszteradat frissítve
- 2026. július 28.
Ki vehet részt
- Életkor
- 6 év – 18 év
- Nem
- Nők és férfiak
- Egészséges önkéntesek
- Nem vehetnek részt
A regiszter feltételei, szó szerint
Mind a 8 feltétel megnézéseKevesebb
Ki vehet részt
- Participants between the ages of 6 to 18 with genetically confirmed congenital or infantile-onset myotonic dystrophy type 1 (DM1)
- Participants willing to stay on stable medication from the day of screening to end of study
Ki nem vehet részt
- Failure to provide signed informed consent by participant or designated decision maker (i.e. parent, legal guardian or power of attorney)
- Failure to provide signed informed consent by parents/caregivers for dyad participation
- Insufficient English language skills to complete required assessments and questionnaires
- Participant is non-verbal
- Participant is not a resident of Canada, due to risk of attrition
- Patients for whom - in the opinion of the investigator - it would not be safe to participate in the study
Ennek a vizsgálatnak nincs helyszíne itt: Egyesült Államok.
Egy országban van helyszíne. Válassz országot, hogy lásd a helyszíneket.
A vizsgálatról
Mit akar kideríteni a vizsgálat
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and heart symptoms of DM1. Additionally, information from the collection of biological samples and wearable devices (accelerometer, EEG headband and ECG chest strap) will be used to identify brain-heart biomarkers and outcome measures for use in future research and trials.
A teljes eredeti leírásKevesebb
Researchers will compare the results of physical and cognitive assessments for each participant to assessments from baseline after 10 weeks of weekly music sessions. Qualitative measures (questionnaires and focus groups) will inform the feasibility of this intervention for this population. The main questions this study aims to answer are:
* Are weekly music education sessions feasible for children with DM1? * Are weekly music education sessions tolerable for children with DM1?
Participants will:
* Attend 45-minute-long music sessions once weekly for 10 weeks. * Attend two clinic visits for cognitive and physical assessments. * Provide blood, saliva, stool and urine samples. * Use wearable devices both at-home and during music sessions. * Parents/caregivers of participants will complete questionnaires and participate in three focus groups.
Progression from feasibility study to a full-scale clinical trial will be informed by four progression criteria:
1. The feasibility of attendance, as assessed by attendance rate to 10 music sessions (≥ 60%) 2. Feasibility of attendance, as rated by parents/caregivers of participants (≥60% rate "extremely" or "very" practical to attend) 3. Attrition rate of the study, as determined by percentage of participants who complete the study (≥ 60%) 4. Overall satisfaction, as rated by parents/caregivers of participants (≥60% rate "very satisfied" or "satisfied")
A vizsgálat csoportja
- Music InterventionA vizsgált kezelés
All participants will receive weekly music and movement education sessions.
Kezelés: Music and movement education
Adatok és források
- Regiszteradat
- Szerepel a ClinicalTrials.gov-on.
- Publikációk
- A regiszteradat frissítve
- 2026. július 28.