Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

NCT ID: NCT02448667

Last Updated: 2024-02-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-01-31

Study Completion Date

2021-05-25

Brief Summary

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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.

Detailed Description

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It has recently been documented how patients with GSDIIIa have a moderate to severely reduced exercise capacity, and that exercise induces muscle pain and cramps. These symptoms are caused by the inability to mobilize skeletal muscle glycogen and are most likely the consequence of a severe energy deficiency within muscles. The study changed the phenotype of GSDIIIa, to include exercise-induced symptoms, which is a typical presentation in other metabolic myopathies. It also documented that exercise capacity was significantly improved while exercise-induced muscular symptoms were relieved by an intravenous glucose infusion. Based on these findings, this study wishes to investigate if oral ingestion of sucrose has the same effects on work capacity on a larger number of patients, in a randomized, placebo-controlled, cross-over setup. Ingestion of sucrose has the potential to be an effective, cheap and easily accessible dietary treatment of muscular symptoms in GSDIIIa.

Conditions

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Glycogen Storage Disease Type III

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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FAXE Kondi - a sugary soft-drink

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

Group Type EXPERIMENTAL

FAXE Kondi

Intervention Type DIETARY_SUPPLEMENT

Sucrose and glucose containing softdrink

FAXE Kondi Free - a sugarfree soft-drink

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.

Group Type PLACEBO_COMPARATOR

Faxe Kondi Free

Intervention Type 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.

Interventions

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FAXE Kondi

Sucrose and glucose containing softdrink

Intervention Type DIETARY_SUPPLEMENT

Faxe Kondi Free

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.

Intervention Type DIETARY_SUPPLEMENT

Eligibility Criteria

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Inclusion Criteria

* Genetically and/or biochemically verified GSDIIIa.
* 18 years or older.

Exclusion Criteria

* 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.
* Other conditions of the joints or skeletal muscle such as arthritis or sprains. If the condition is expected to resolve before the study inclusion period is stopped, the subject may be included at a later time.
* 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.
* Blood donation one month or less prior to inclusion.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Rigshospitalet, Denmark

OTHER

Sponsor Role lead

Responsible Party

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Astrid Emilie Buch

BSc

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Astrid E Buch, BSc Medicine

Role: PRINCIPAL_INVESTIGATOR

Copenhagen Neuromuscular Center

Locations

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Copenhagen Neuromuscular Center, department 3342, Rigshospitalet

Copenhagen, Capital Region, Denmark

Site Status

Countries

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Denmark

References

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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.

Reference Type BACKGROUND
PMID: 20631546 (View on PubMed)

Van Hoof F, Hers HG. The subgroups of type 3 glycogenosis. Eur J Biochem. 1967 Oct;2(3):265-70. doi: 10.1111/j.1432-1033.1967.tb00134.x. No abstract available.

Reference Type BACKGROUND
PMID: 5235982 (View on PubMed)

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.

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Reference Type BACKGROUND
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Reference Type BACKGROUND
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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.

Reference Type BACKGROUND
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Reference Type BACKGROUND
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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.

Reference Type BACKGROUND
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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.

Reference Type BACKGROUND
PMID: 16970212 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 24741154 (View on PubMed)

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.

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PMID: 23891579 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17828671 (View on PubMed)

EFSA ANS Panel (EFSA Panel on Food Additives and Nutrient Sources added to food), 2013. Scientific Opinion on the re-evaluation of aspartame (E 951) as a food additive. EFSA Journal 2013;11(12):3496, 263 pp. doi:10.2903/j.efsa.2013.3496

Reference Type 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

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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

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Related Links

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http://ec.europa.eu/food/fs/sc/scf/out52_en.pdf

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

Other Identifiers

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H-4-2014-014

Identifier Type: -

Identifier Source: org_study_id

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