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

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ClinicalTrials.gov Identifier: NCT04634682
Recruitment Status : Recruiting
First Posted : November 18, 2020
Last Update Posted : November 18, 2020
Sponsor:
Information provided by (Responsible Party):
Myogem Health Company, S.L.

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Myotonic Dystrophy 1 Dietary Supplement: MYODM Not Applicable

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Assessing the Effect of the MYODM Food Supplement on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
Actual Study Start Date : November 9, 2020
Estimated Primary Completion Date : August 2021
Estimated Study Completion Date : September 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Fatigue

Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Change in Individualized Neuromuscular Quality of Life (INQoL) Mean Scores [ Time Frame: Screening, Month 3, Month 6 ]
    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.

  2. Change in Individualized Short Form-36 (SF-36) Mean Scores [ Time Frame: Screening, Month 3, Month 6 ]
    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.

  3. Change in Epworth Sleepiness Scale (ESS) Scores [ Time Frame: Screening, Month 3, Month 6 ]
    ESS score range is 0-24; lower ESS scores indicate less daytime sleepiness; higher ESS scores indicate more severe sleepiness


Secondary Outcome Measures :
  1. Change in Physical activity and daytime sleepiness measured with GeneActiv actometer [ Time Frame: Screening, Baseline, Month 6 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04634682


Locations
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Spain
Hospital Universitario Donostia Recruiting
San Sebastián, Guipuzkoa, Spain, 20014
Contact: Roberto Fernandez Torron    943 00 70 27    roberto.fernandeztorron@osakidetza.eus   
Sponsors and Collaborators
Myogem Health Company, S.L.
More Information
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Responsible Party: Myogem Health Company, S.L.
ClinicalTrials.gov Identifier: NCT04634682    
Other Study ID Numbers: RFT-MYO-2020-01
First Posted: November 18, 2020    Key Record Dates
Last Update Posted: November 18, 2020
Last Verified: November 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myotonic Dystrophy
Disorders of Excessive Somnolence
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
 Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Wake Disorders
Mental Disorders