McArdle Disease Clinical Trial
— GSDVOfficial title:
Oral Ketone Body Supplementation in Patients With McArdle Disease
Verified date | October 2020 |
Source | Rigshospitalet, Denmark |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 8 |
Est. completion date | October 1, 2020 |
Est. primary completion date | October 1, 2020 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 18 Years and older |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
Denmark | Copenhagen Neuromuscular Center | Copenhagen |
Lead Sponsor | Collaborator |
---|---|
Rigshospitalet, Denmark |
Denmark,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Heart rate | Change between visit 1 and 2 in mean heart rate during constant load cycling exercise (40 minute submaximal cycle test) | 30 minuts x 2 | |
Secondary | Ketone metabolism | Change between visit 1 and 2, measured with stable isotope technique during the constant load cycling test | 150 minuts x 2 | |
Secondary | Carbohydrate metabolism | Change between visit 1 and 2, measured with stable isotope technique during the constant load cycling test | 150 minuts x 2 | |
Secondary | Fat metabolism | Change between visit 1 and 2, measured with stable isotope technique during the constant load cycling test | 150 minuts x 2 | |
Secondary | Indirect calorimetry | Changes in oxidation rates at visit 1 and visit 2, measured via indirect calorimetry before and during exercise | 30 minuts x 2 | |
Secondary | Perceived exertion | Changes in perceived exertion beween visit 1 and 2 during the cycle test | 30 minuts x 2 | |
Secondary | Blood ketone | Changes in ketone bodies in the blood between visit 1 and 2, measured at both before, during and at end exercise. | 150 minuts x 2 | |
Secondary | Insulin | Insulin changes between visit 1 and 2 measured before, during and at end exercise. | 30 minuts x 2 | |
Secondary | Metabolites | changes between visit 1 and 2 | 30 minuts x 2 |
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