UnknownNot Applicable

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Denmark60 participantsStarted 2016-01

Plain-language summary

This study aims to characterize the pathophysiological mechanisms of 21 different metabolic myopathies. The study will focus on exercise capacity and the metabolic derangement during exercise.

Who can participate

Age range

15 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Men and women with genetically and/or biochemically verified LCHAD Deficiency * Healthy controls needs to be healthy to be included, evaluated by the investigator. Exclusion Criteria: * Competing conditions at risk of compromising the results of the study. * Pregnancy or breastfeeding * Cardiac or pulmonary disease contraindicating peak exercise testing or strenuous exercise. * Inability to understand the purpose of the trial or corporate for the conduction of the experiments. * Veins that are too difficult to puncture for blood sampling or insertion of intravenous catheters, evaluated by the investigator * Moderate to severe muscle weakness, that prevents the subject completing 10 minutes of cycle-ergometry exercise at 60-70 % of VO2peak, evaluated by the investigator. * Participation in other trials that may interfere with the results. * Intake of medications that may interfere with the results or may compromise exercise performance, as evaluated by the investigators. * Donation of blood within 30 days prior to conduction of the tests on test day 1.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Total fat oxidation (rate of disappearance) during prolonged moderate intensity exercise. Measured as micro mol per kg per minute.

Timeframe: Measured at at time (minutes) -120, -20, -10, 0, 10, 20, 30, 40, 50, 60. Zero is the indicatet start of 1 hour cycling test. Measured on day 2 of participation

Trial details

NCT IDNCT02635269

SponsorRigshospitalet, Denmark

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-08

View on ClinicalTrials.gov More Metabolism, Inborn Errors trials