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

NCT04634682 | Completed

• 1 other identifier: RFT-MYO-2020-01
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Myotonic Dystrophy 1

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

• Change in Physical activity and daytime sleepiness measured with GeneActiv actometer

  Screening, Baseline, Month 6

Study Arms (2)

MYODM

EXPERIMENTAL

MYODM, three times a day, orally

Dietary Supplement: MYODM

No intervention

NO INTERVENTION

Patients will follow the same evaluation schedule but will not receive MYODM

Interventions

MYODM DIETARY_SUPPLEMENT

Formulated composition containing Theobroma cacao supplemented with caffeine

MYODM

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able to provide informed consent
• Genetically proven DM1
• Able to walk independently

You may not qualify if:

• 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.

Sponsors & Collaborators

• Myogem Health Company, S.L.: lead

Study Sites (1)

Hospital Universitario Donostia

San Sebastián, Guipuzkoa, 20014, Spain

Location

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2020
• First Posted: November 18, 2020
• Study Start: November 9, 2020
• Primary Completion: November 15, 2021
• Study Completion: December 3, 2021
• Last Updated: April 6, 2022

Record last verified: 2022-04

Locations

ES (1)