Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

NCT02448667 | Completed

• 1 other identifier: H-4-2014-014
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Glycogen Storage Disease Type III

Outcome Measures

Primary Outcomes (1)

• maximal work capacity

  Area Under the Curve (AUC) = resistance times duration of workout

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

Secondary Outcomes (6)

• Peak oxygen consumption

  After up to 1 hour of cycling on the 2nd and 4th day.

• Peak workload

  After up to 1 hour of cycling on the 2nd and 4th day.

• Peak respiratory exchange ratio

  After up to 1 hour of cycling on the 2nd and 4th day.

• p-lactate

  measured at rest and max on day 1, and before first dose of soft drink, before exercise and every 10 minutes during exercise at day 2 and 4.

• Heart rate

  Continously during the cycle test (max. 1 hour) on the 2nd and 4th day

Other Outcomes (11)

• Respiratory exchange ratio, RER

  measured continously during the exercise test day 2 and 4.

• p-glucose

  measured at rest and max on day 1, and before first dose of soft drink, before exercise and every 10 minutes during exercise at day 2 and 4.

• Pain

  Assessed on days 3 and 5 of the trial

Study Arms (2)

FAXE Kondi - a sugary soft-drink

EXPERIMENTAL

100 ml FAXE Kondi (10 grams of carbohydrates per 100 ml) is ingested every ten minutes during exercise plus 400 ml before exercise start.

Dietary Supplement: FAXE Kondi

FAXE Kondi Free - a sugarfree soft-drink

PLACEBO COMPARATOR

100 ml FAXE Kondi Free (0 grams of carbohydrates per 100 ml) is ingested every ten minutes during exercise plus 400 ml before exercise start.

Dietary Supplement: Faxe Kondi Free

Interventions

FAXE Kondi DIETARY_SUPPLEMENT

Sucrose and glucose containing softdrink

FAXE Kondi - a sugary soft-drink

Faxe Kondi Free DIETARY_SUPPLEMENT

Diet softdrink with artificial sweeteners aspartame and acesulfame potassium. Both sweeteners are approved for use as food additives in the European Union and by the FDA. Aspartame metabolism is well understood and normal doses does not affect plasma concentrations of lipids, amino acids, glucose levels, key regulatory hormones or skeletal muscle metabolism. Acesulfame Potassium is not metabolized in humans and is excreted as the parent compound in urine. Since the two artificial sweeteners does not affect skeletal muscle metabolism or blood glucose levels, and both compounds have a well documented safety profiles, FAXE Kondi Free is considered to be an ideal placebo soft drink in this study.

FAXE Kondi Free - a sugarfree soft-drink

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Genetically and/or biochemically verified GSDIIIa.
• years or older.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Rigshospitalet, Denmark: lead

Study Sites (1)

Copenhagen Neuromuscular Center, department 3342, Rigshospitalet

Copenhagen, Capital Region, 2100, Denmark

Location

Related Publications (16)

• Kishnani PS, Austin SL, Arn P, Bali DS, Boney A, Case LE, Chung WK, Desai DM, El-Gharbawy A, Haller R, Smit GP, Smith AD, Hobson-Webb LD, Wechsler SB, Weinstein DA, Watson MS; ACMG. Glycogen storage disease type III diagnosis and management guidelines. Genet Med. 2010 Jul;12(7):446-63. doi: 10.1097/GIM.0b013e3181e655b6.

  PMID: 20631546 BACKGROUND

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  PMID: 5235982 BACKGROUND

• Coleman RA, Winter HS, Wolf B, Gilchrist JM, Chen YT. Glycogen storage disease type III (glycogen debranching enzyme deficiency): correlation of biochemical defects with myopathy and cardiomyopathy. Ann Intern Med. 1992 Jun 1;116(11):896-900. doi: 10.7326/0003-4819-116-11-896.

  PMID: 1580445 BACKGROUND

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  PMID: 23507172 BACKGROUND

• Haller RG, Vissing J. Spontaneous "second wind" and glucose-induced second "second wind" in McArdle disease: oxidative mechanisms. Arch Neurol. 2002 Sep;59(9):1395-402. doi: 10.1001/archneur.59.9.1395.

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• Dupont WD, Plummer WD Jr. Power and sample size calculations. A review and computer program. Control Clin Trials. 1990 Apr;11(2):116-28. doi: 10.1016/0197-2456(90)90005-m.

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• Preisler N, Laforet P, Madsen KL, Hansen RS, Lukacs Z, Orngreen MC, Lacour A, Vissing J. Fat and carbohydrate metabolism during exercise in late-onset Pompe disease. Mol Genet Metab. 2012 Nov;107(3):462-8. doi: 10.1016/j.ymgme.2012.08.019. Epub 2012 Aug 31.

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• Borg G. Perceived exertion as an indicator of somatic stress. Scand J Rehabil Med. 1970;2(2):92-8. No abstract available.

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• Coyle EF. Carbohydrate supplementation during exercise. J Nutr. 1992 Mar;122(3 Suppl):788-95. doi: 10.1093/jn/122.suppl_3.788.

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• Maki DG, Kluger DM, Crnich CJ. The risk of bloodstream infection in adults with different intravascular devices: a systematic review of 200 published prospective studies. Mayo Clin Proc. 2006 Sep;81(9):1159-71. doi: 10.4065/81.9.1159.

  PMID: 16970212 BACKGROUND

• Chattopadhyay S, Raychaudhuri U, Chakraborty R. Artificial sweeteners - a review. J Food Sci Technol. 2014 Apr;51(4):611-21. doi: 10.1007/s13197-011-0571-1. Epub 2011 Oct 21.

  PMID: 24741154 BACKGROUND

• Marinovich M, Galli CL, Bosetti C, Gallus S, La Vecchia C. Aspartame, low-calorie sweeteners and disease: regulatory safety and epidemiological issues. Food Chem Toxicol. 2013 Oct;60:109-15. doi: 10.1016/j.fct.2013.07.040. Epub 2013 Jul 23.

  PMID: 23891579 BACKGROUND

• Magnuson BA, Burdock GA, Doull J, Kroes RM, Marsh GM, Pariza MW, Spencer PS, Waddell WJ, Walker R, Williams GM. Aspartame: a safety evaluation based on current use levels, regulations, and toxicological and epidemiological studies. Crit Rev Toxicol. 2007;37(8):629-727. doi: 10.1080/10408440701516184.

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  BACKGROUND

• Harris RA. Carbohydrate metabolism I: Major metabolic pathways and their control. In: Devlin TM, ed. Textbook of biochemistry with clinical correlations, 6th ed Wiley-Liss, 2006:581-635

  BACKGROUND

• DiMauro S, Hays AP, Tsujino S. Metabolic Disorders Affecting Muscle. In: Engel AG, Franzini-Armstrong C, eds. Myology, 3rd ed McGraw-Hill, 2004:1535-1558

  BACKGROUND

Related Links

• Scientific Committee on Food. Re-evaluation of acesulfame K with reference to the previous SCF opinion of 1991.

MeSH Terms

Conditions

Glycogen Storage Disease Type III

Condition Hierarchy (Ancestors)

Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Astrid E Buch, BSc Medicine

  Copenhagen Neuromuscular Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: BSc

Study Record Dates

• First Submitted: May 15, 2015
• First Posted: May 19, 2015
• Study Start: January 1, 2015
• Primary Completion: April 17, 2017
• Study Completion: May 25, 2021
• Last Updated: February 26, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1)