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

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

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.

Study Overview

• Status: Completed
• Conditions: Myotonic Dystrophy 1
• Intervention / Treatment: Dietary supplement: MYODM

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Spain
  • Guipuzkoa
    • San Sebastián, Guipuzkoa, Spain, 20014
      • HOSPITAL UNiVERSITARIO DONOSTIA

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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	Time Frame
Change in Physical activity and daytime sleepiness measured with GeneActiv actometer	Screening, Baseline, Month 6

Collaborators and Investigators

• Sponsor: Myogem Health Company, S.L.

Study record dates

Study Major Dates

• Study Start (Actual): November 9, 2020
• Primary Completion (Actual): November 15, 2021
• Study Completion (Actual): December 3, 2021

Study Registration Dates

• First Submitted: September 25, 2020
• First Submitted That Met QC Criteria: November 17, 2020
• First Posted (Actual): November 18, 2020

Study Record Updates

• Last Update Posted (Actual): April 6, 2022
• Last Update Submitted That Met QC Criteria: April 5, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Muscular Disorders, Atrophic; Heredodegenerative Disorders, Nervous System; Muscular Dystrophies; Myotonic Disorders; Myotonic Dystrophy; Disorders of Excessive Somnolence
• Other Study ID Numbers: RFT-MYO-2020-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No