Glycogen Storage Disease Type II Clinical Trial
Official title:
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
Verified date | October 2019 |
Source | Rigshospitalet, Denmark |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 60 |
Est. completion date | December 2020 |
Est. primary completion date | August 2020 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 15 Years to 80 Years |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
Denmark | Neuromuscular Research Unit, 3342 | Copenhagen |
Lead Sponsor | Collaborator |
---|---|
Rigshospitalet, Denmark |
Denmark,
Liang WC, Nishino I. State of the art in muscle lipid diseases. Acta Myol. 2010 Oct;29(2):351-6. Review. — View Citation
Oldfors A, DiMauro S. New insights in the field of muscle glycogenoses. Curr Opin Neurol. 2013 Oct;26(5):544-53. doi: 10.1097/WCO.0b013e328364dbdc. Review. — View Citation
Ørngreen MC, Dunø M, Ejstrup R, Christensen E, Schwartz M, Sacchetti M, Vissing J. Fuel utilization in subjects with carnitine palmitoyltransferase 2 gene mutations. Ann Neurol. 2005 Jan;57(1):60-6. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Total fat oxidation (rate of disappearance) during prolonged moderate intensity exercise. Measured as micro mol per kg per minute. | Measured using stable isotope technique ([U-13C]-palmitate, [1,1,2,3,3-2H5]-glycerol and [6.6-2H2]-glucose ) and indirect calorimetry. | 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 | |
Secondary | Maximal oxidative capacity (mL*kg-1*minutes-1) | Oxidative capacity is measured continuously during a maximum exercise test on the first day of the trial. The test lasts about 15 minutes (when the participant is fully exhausted). The maximal oxidative capacity is the peak measure during the test. | Day 1 of participation | |
Secondary | Endurance measured as minutes:seconds | During 1 hour cycling test 1 and 1 hour cycling test 2 on day 2 and 4 of participation. The test will last for 1 hour or until exhaustion. Endurance will be defined as the total exercise duration. | Day 2 and 4 of participation | |
Secondary | Heart rate (HR) during prolonged exercise, with and without blood sugar maintained by an infusion of a 10% glucose solution. Measured in beats per minute. | During 1 hour cycling at 55-65% of VO2max the heart rate is measured every 2 minuts. | During 1 hour cycling test 1 and 1 hour cycling test 2 on day 2 and 4 of participation. | |
Secondary | Oxygen consumption rate (VO2) during prolonged exercise, with and without blood sugar maintained by an infusion of a 10% glucose solution. Measured in mL. | During 1 hour cycling at 55-65% of VO2max the oxygen consumption rate is continuously measured. | During 1 hour cycling test 1 and 1 hour cycling test 2 on day 2 and 4 of participation. | |
Secondary | Self-evaluated exertion (Rate of Perceived Exertion (RPE)) during prolonged exercise, with and without blood sugar maintained by an infusion of a 10% glucose solution. Measured using a Borg-scale (6-20). | During 1 hour cycling at 55-65% of VO2max the participant is asked to rate their exertion every 2 minutes. | During 1 hour cycling test 1 and 1 hour cycling test 2 on day 2 and 4 of participation. | |
Secondary | Maximal workload capacity. Measured in watt | The participant will be cycling at incremental workload until exhaustion. The test lasts about 15 minutes (when the participant is fully exhausted). The maximal workload is the peak measure at the end of the test. | Day 1 of participation | |
Secondary | Total glucose oxidation (rate of disappearance) during prolonged moderate intensity exercise. Measured as micro mol per kg per minute. | Measured using stable isotope technique ([U-13C]-palmitate, [1,1,2,3,3-2H5]-glycerol and [6.6-2H2]-glucose ) and indirect calorimetry. | 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 |
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