Skip to main content
OpenTrials
Completed

NCT Number: NCT02448667

Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Copenhagen Neuromuscular Center, department 3342, Rigshospitalet

Copenhagen, Capital Region, 2100, Denmark

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

FAXE Kondi

Dietary Supplement

Sucrose and glucose containing softdrink

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.

Primary outcomes

  1. maximal work capacity

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

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

Secondary outcomes

  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.

    Analysis of blood sample

  5. Heart rate

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

    pulsemonitoring

  6. Borg score

    Time frame: Measured periodically during the cycle test (max. 1 hour) on the 2nd and 4th day

    Rate of percieved exertion

Other outcomes

  1. Respiratory exchange ratio, RER

    Time frame: measured continously during the exercise test day 2 and 4.

    VO2/VCO2

  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.

    Analysis of blood sample

  3. Pain

    Time frame: Assessed on days 3 and 5 of the trial

    Pain assessed on a visual analog scale (VAS) with a scale of 0 to 10 cm

  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-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.

    Analysis of blood sample

  8. 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.

    analysis of blood sample

  9. 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.

    analysis of blood sample

  10. 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.

    analysis of blood sample

  11. Hypoglycemic episodes

    Time frame: 2 hour observation after each of the two exercise test.

    Clinical observation as well as blood glucose levels monitored during exercise tests

Sponsors and collaborators

Lead sponsor

Rigshospitalet, Denmark

Other

Registry information

Important dates

Study start
2015
Primary completion
2017
Study completion
2021
First posted
May 19, 2015
Registry last updated
Feb 26, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.