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Trial record 29 of 662 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Diseases"

Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

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ClinicalTrials.gov Identifier: NCT02448667
Recruitment Status : Recruiting
First Posted : May 19, 2015
Last Update Posted : January 20, 2017
Sponsor:
Information provided by (Responsible Party):
Astrid Emilie Buch, Rigshospitalet, Denmark

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.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type III Dietary Supplement: FAXE Kondi Dietary Supplement: Faxe Kondi Free Not Applicable

Detailed Description:
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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies
Study Start Date : January 2015
Estimated Primary Completion Date : November 2017
Estimated Study Completion Date : February 2018


Arm Intervention/treatment
Experimental: 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.
Dietary Supplement: FAXE Kondi
Sucrose and glucose containing softdrink

Placebo Comparator: 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.
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.




Primary Outcome Measures :
  1. maximal work capacity [ Time Frame: After up to 1 hour of bicycling on the 2nd and 4th day. ]
    Area Under the Curve (AUC)


Secondary Outcome Measures :
  1. Peak oxygen consumption [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (VO2peak)

  2. Peak workload [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (Wpeak)

  3. Peak respiratory exchange ratio [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (RER)

  4. p-lactate [ Time Frame: 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. ]
  5. Heart rate [ Time Frame: Continously during the cycle test (max. 1 hour) on the 2nd and 4th day ]
  6. Borg score [ Time Frame: Measured periodically during the cycle test (max. 1 hour) on the 2nd and 4th day ]

Other Outcome Measures:
  1. Respiratory exchange ratio, RER [ Time Frame: measured continously during the exercise test day 2 and 4. ]
  2. p-glucose [ Time Frame: 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. ]
  3. Pain [ Time Frame: Assessed on days 3 and 5 of the trial ]
    Visual analog scale (VAS)

  4. Fatigue [ Time Frame: Assessed on days 3 and 5 of the trial ]
    Fatigue Severity Score (FSS)

  5. p-Creatine kinase [ Time Frame: measured on day 1, 3 and 5. ]
    To asses muscle damage

  6. p-myoglobin [ Time Frame: measured on day 1, 3 and 5. ]
    To asses muscle damage

  7. p-free fatty acids [ Time Frame: measured before first dose of soft drink, before exercise and every 10 minutes during exercise at day 2 and 4. ]
  8. p-ketone bodies [ Time Frame: 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. ]
  9. p-ammonia [ Time Frame: measured at rest and max on day 1, and before exercise, at 10 minutes, 20 min of exercise and at max on day 2 and 4. ]
  10. p-insulin [ Time Frame: measured at rest and max on day 1 and before exercise and every 10 minutes during exercise at day 2 and 4. ]
  11. p-glucagon [ Time Frame: measured at rest and max on day 1, and before exercise, at 10 minutes, 20 min of exercise and at max on day 2 and 4. ]
  12. p-catecholamines [ Time Frame: measured at rest and max on day 1, and before exercise, at 10 minutes, 20 min of exercise and at max on day 2 and 4. ]
  13. p-sodium [ Time Frame: before exercise on day 2 and 4 ]
    To eliminate electrolyt status as a confounding factor

  14. p-potassium [ Time Frame: before exercise on day 2 and 4 ]
    To eliminate electrolyt status as a confounding factor

  15. Hypoglycemic episodes [ Time Frame: 2 hour observation after each of the two exercise test. ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02448667


Contacts
Contact: Astrid E Buch, BSc Medicine +45 35 45 61 35 astrid.emilie.buch.02@regionh.dk
Contact: Nicolai Preisler, MD +45 35 45 61 26 nicolai.preisler@regionh.dk

Locations
Denmark
Copenhagen Neuromuscular Center, department 3342, Rigshospitalet Recruiting
Copenhagen, Region hovedstaden, Denmark, 2100
Contact: Astrid E Buch, MD    +45 35 45 61 35    astrid.emilie.buch.02@regionh.dk   
Contact: Nicolai Preisler, MD    +45 35 45 61 26    nicolai.preisler@regionh.dk   
Sponsors and Collaborators
Rigshospitalet, Denmark
Investigators
Principal Investigator: Astrid E Buch, BSc Medicine Copenhagen Neuromuscular Center

Additional Information:
Publications:
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
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
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

Responsible Party: Astrid Emilie Buch, BSc, Rigshospitalet, Denmark
ClinicalTrials.gov Identifier: NCT02448667     History of Changes
Other Study ID Numbers: H-4-2014-014
First Posted: May 19, 2015    Key Record Dates
Last Update Posted: January 20, 2017
Last Verified: January 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Glycogen Storage Disease
Glycogen Storage Disease Type III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases