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

Myotonic Dystrophy 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04634682
• Other study ID #: RFT-MYO-2020-01
• Status: Completed
• Phase: N/A
• Start date: November 9, 2020
• Est. completion date: December 3, 2021

Study information

• Verified date: April 2022
• Source: Myogem Health Company, S.L.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: December 3, 2021
• Est. primary completion date: November 15, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Disorders of Excessive Somnolence
• Myotonic Dystrophy
• Myotonic Dystrophy 1

Intervention

Dietary Supplement:
• MYODM
Formulated composition containing Theobroma cacao supplemented with caffeine

Locations

Country	Name	City	State
Spain	Hospital Universitario Donostia	San Sebastián	Guipuzkoa

Sponsors (1)

Lead Sponsor	Collaborator
Myogem Health Company, S.L.

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	Change in Physical activity and daytime sleepiness measured with GeneActiv actometer		Screening, Baseline, Month 6