Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1

Study Overview
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Brief Summary
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The purpose of this study is to determine whether MYODM (formulated composition containing Theobroma cacao supplemented with caffeine (caffeine/theobromine ratio1/1.85, w/w) is effective in the treatment of excessive daytime sleepiness due to myotonic dystrophy type 1 (DM1) and improves the quality of life of these patients.

Detailed Description
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Detailed Description: Myotonic Dystrophy type I (DM1) is the most common form of adult muscular dystrophy, affecting 1 in 8000 individuals. It is an autosomal dominant disorder with multisystemic involvement of multiple organs and tissues, mainly brain, heart, endocrine system, eyes and both smooth and skeletal muscles. Hypersomnolence is one of the most frequently reported symptoms in patients with DM1 and often lead to handicap such as cessation of employment and withdrawal from social activities. The present project is a 6 month randomized study to assess the effect of MYODM on fatigue, hypersomnia and quality of life in DM1 patients.The patients will be randomized to one of the two study arms. The active arm will receive the MYODM treatment and the control arm will not but both will follow the same evaluation program. Patients will come to the center every 3 months for evaluations.

Official Title
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Assessing the Effect of the MYODM Food Supplement on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1

Conditions
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Myotonic Dystrophy 1

Intervention / Treatment
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* Dietary Supplement: MYODM


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Able to provide informed consent Genetically proven DM1 Able to walk independently Exclusion Criteria: Regular intake of plant stanols or other nutritional supplement Co-morbidity interfering with the interventions or possibly influencing outcomes. Participation in another clinical trial at the same time Unable to complete study questionnaires.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MYODM<br>MYODM, three times a day, orally | Dietary Supplement: MYODM<br>* Formulated composition containing Theobroma cacao supplemented with caffeine<br>|
| No Intervention: No intervention<br>Patients will follow the same evaluation schedule but will not receive MYODM | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Individualized Neuromuscular Quality of Life (INQoL) Mean Scores | Scores from the self-administered INQoL questionnaire will be compared at the start of the study (Month 0) and at the end (Month 6) among the MYODM treated group and the control group. Scores range from 0-100, with 100 being a better outcome. | Screening, Month 3, Month 6 | 
| Change in Individualized Short Form-36 (SF-36) Mean Scores | Scores from the self-administered SF-36 questionnaire will be measured at the start of the study (Month 0), and at the end (Month 6) among patients in the MYODM-treated group and control group. Mean scores range from 0 (minimum) - 100 (maximum) with higher mean scores reflecting better outcomes. | Screening, Month 3, Month 6 | 
| Change in Epworth Sleepiness Scale (ESS) Scores | ESS score range is 0-24; lower ESS scores indicate less daytime sleepiness; higher ESS scores indicate more severe sleepiness | Screening, Month 3, Month 6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Physical activity and daytime sleepiness measured with GeneActiv actometer |  | Screening, Baseline, Month 6 |