CompletedNot Applicable

Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

Denmark6 participantsStarted 2015-01

Plain-language summary

Patients suffering from the metabolic myopathy Glycogen Storage Disease type IIIa (GSDIIIa) have a problem releasing sugar stored in cells that is needed for energy production. This causes several systemic impairments, but only recently have the exercise-related symptoms in the muscles been examined. A previous study showed signs that intravenous infusion of glucose relieves some of these symptoms. The purpose of this study is to investigate in a randomized and placebo-controlled fashion whether oral ingestion of sugar can alleviate muscular symptoms in patients with GSDIIIa.

Who can participate

Age range

18 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: * Genetically and/or biochemically verified GSDIIIa. * 18 years or older. Exclusion Criteria: * Clinically significant cardiac or pulmonary disease. * Pregnancy or lactation. * Severe mental disorders or participants that are in other ways unable to understand the purpose of the trials. * Subjects where the investigator assess that it is not possible or very difficult to place an intravenous catheters. * Other conditions of the joints or skeletal muscle such as arthritis or sprains. If the condition is expected to resolve before the study inclusion period is stopped, the subject may be included at a later time. * Moderate to severe muscle weakness, where the participants are not expected to complete 10 minutes of cycle-ergometry exercise at 70 % of VO2peak. * Verified diabetes. * Participation in other clinical trials that may interfere with the results. * Medications that may interfere with the results or increase the risk of bleeding. * Blood-clotting or bleeding disorders. * Blood donation one month or less prior to inclusion.

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

maximal work capacity

Timeframe: After up to 1 hour of bicycling on the 2nd and 4th day.

Trial details

NCT IDNCT02448667

SponsorRigshospitalet, Denmark

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-04-17

View on ClinicalTrials.gov More Glycogen Storage Disease Type III trials