Myotonic Dystrophy 1NCT04634682
Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
A study testing MYODM in people with Myotonic Dystrophy 1, run by Myogem Health Company, S.L. at 1 site in Spain.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- MYODM
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteSpain
- Study dates
- 9 Nov 2020to 3 Dec 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to provide informed consent
- Genetically proven DM1
- Able to walk independently
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- MYODM
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MYODMDietary supplement
Formulated composition containing Theobroma cacao supplemented with caffeine
| Study group | Role | What they receive |
|---|---|---|
| MYODM | Experimental | MYODM, three times a day, orally Receives: Dietary Supplement: MYODM |
| No intervention | No intervention | Patients will follow the same evaluation schedule but will not receive MYODM |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Measured: Screening, Month 3, Month 6
Secondary outcomes (1)
Change in Physical activity and daytime sleepiness measured with GeneActiv actometer
Measured: Screening, Baseline, Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2020
- Study start9 Nov 2020
- Primary completion15 Nov 2021
- Study completion3 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Myogem Health Company, S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04634682
- Sponsor's protocol ID
- RFT-MYO-2020-01
- Status last verified
- Apr 2022
- First submitted
- 25 Sep 2020
- Last updated
- 6 Apr 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessing the Effect of the MYODM Food Supplement on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT04634682
- Acronym / sponsor ID
- RFT-MYO-2020-01
- Conditions
- Myotonic Dystrophy 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Myogem Health Company, S.L.Industry (pharma, biotech, devices)
- Study start
- 9 Nov 2020 (actual)
- Primary completion
- 15 Nov 2021 (actual)
- Study completion
- 3 Dec 2021 (actual)
- First posted
- 18 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MYODM | Experimental | Dietary Supplement: MYODMMYODM, three times a day, orally |
| No intervention | No intervention | –Patients will follow the same evaluation schedule but will not receive MYODM |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Able to provide informed consent
- Genetically proven DM1
- Able to walk independently
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MYODMFormulated composition containing Theobroma cacao supplemented with caffeine | Dietary supplement | MYODM |
Primary outcome
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.
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.
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
1 secondary outcome
Change in Physical activity and daytime sleepiness measured with GeneActiv actometer
Time frame: Screening, Baseline, Month 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MYODM in people with Myotonic Dystrophy 1, run by Myogem Health Company, S.L. at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Individualized Neuromuscular Quality of Life (INQoL) Mean Scores (time frame: Screening, Month 3, Month 6). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2022.