CompletedNot Applicable

Oral Ketone Body Supplementation in Patients With McArdle Disease

Denmark8 participantsStarted 2019-05-06

Plain-language summary

McArdle disease, glycogen storage disease type V, is a rare metabolic disease. Affected individuals are unable to utilize sugar stored as glycogen in muscle. Investigators hypothesize that ketones can be an alternative fuel substrate for skeletal muscle when muscle glycogenolysis is blocked as in McArdle disease. In this study investigators will investigate the immediate effects of an oral supplementation of exogenous ketone bodies (poly-hydroxybuturate) on exercise capacity in patients with metabolic myopathies, compared with a placebo drink.

Who can participate

Age range18 Years

SexALL

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

Inclusion criteria: * Genetically confirmed McArdle disease or healthy control. * Patient is willing and able to provide written informed consent prior to participation. * Patient is ambulatory. Exclusion criteria: * Patient has any prior or current medical conditions that, in the judgment of the Investigator, would prevent the patient from safely participating in and/or completing all study requirements. * Pregnancy or breastfeeding * Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments.

What they're measuring

Heart rate

Timeframe: 30 minuts x 2

Trial details

NCT IDNCT03945370

SponsorRigshospitalet, Denmark

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-10-01

View on ClinicalTrials.gov More McArdle Disease trials