An interventional study of Modified ketogenic diets in McArdle Disease, sponsored by Rigshospitalet, Denmark. Completed at 1 site in Denmark. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2019-05-10.
Sponsored by Rigshospitalet, Denmark · Not applicable, Interventional, and Treatment
McArdle disease, glycogen storage disease type V, is a rare metabolic disease. Affected individuals are unable to utilize sugar stored as glycogen in muscle.
We hypothesize that a modified ketogenic diet could be a potential treatment option, by providing ketones as alternative fuel substrates for working muscle.
In this open interventional pilot study we wish to investigate 3 different modified ketogenic diet regimes, to find an optimal composition of a modified ketogenic diet that ensures adequate degree of ketosis and at the same time is well tolerated for patients with McArdle disease.
Open interventional study to investigate 3 different modified ketogenic diet regimes, to find an optimal composition of a modified ketogenic diet for patients with McArdle disease
McArdle disease, glycogen storage disease type V, is a rare metabolic disease caused by mutations in the PYGM gene resulting in absence of the enzyme muscle phosphorylase. Affected individuals are unable to utilize sugar stored as glycogen in muscle, leading to exercise intolerance, exercise-induced muscle pain, contractures and rhabdomyolysis, which may cause renal failure. Currently, there are no satisfactory treatment options for McArdle disease. Ketones are feasible fuel alternatives to muscle glycogen when muscle glycogenolysis is blocked as in McArdle disease.
A key element of alleviating symptoms in McArdle disease is to provide alternative fuels for energy metabolism. Ketosis can potentially provide alternative fuel substrates by provision of endogenous ketone bodies (KBs) which are desirable fuels for skeletal muscle and brain. Ketosis can be reached by fasting and can be induced by adhering to a modified ketogenic diet, which entails a high-fat, low-carbohydrate diet, which simulates the metabolic effects of fasting.
The optimal modified ketogenic diet composition, that ensures adequate degree of ketosis and at the same time is well tolerated, has not been investigated in patients with McArdle disease. Therefore this pilot study seeks to investigate which of 3 different diets is the most optimal ad effective for patients with McArdle disease. The 3 different diets will have carbohydrate percentages ranging from 5-25%, and fat percentage from 60-80%.
Rigshospitalet, Denmark is the lead sponsor of 1,017 studies on the registry; 183 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
60% fat, 15% protein, 25% carbohydrates
Other: Modified ketogenic diets
70% fat, 15% protein, 15% carbohydrates
Other: Modified ketogenic diets
80% fat, 15% protein, 5% carbohydrates
Other: Modified ketogenic diets
Different diet composition
Heart rate
heart rate during constant load cycling exercise
Time frame: 2 times
Compliance
Dietary diary to evaluate compliance and diet acceptability
Time frame: 3 weeks
indirect calorimetry
Oxidation rates measured via indirect calorimetry during constant load cycling calorimetry
Time frame: 2 times
level of ketosis
Ketone bodies in the blood
Time frame: daily
Safety parameters
Blood samples
Time frame: 2 times
Perceived exertion
Borg scale during constant load cycling
Time frame: 2 times
other blood samples
Cholesterol, fatty acids, hormones, ammonia
Time frame: 2 times
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in May 2019. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Glycogen Storage Disease Type V
Rigshospitalet, Denmark