Back to resultsEnergy Supplements to Improve Exercise Tolerance in Metabolic Myopathies
Primary Purpose
Glycogen Storage Disease Type III
Status
Unknown status
Phase
Not Applicable
Locations
Denmark
Study Type
Interventional
Intervention
FAXE Kondi
Faxe Kondi Free
Sponsored by
About this trial
This is an interventional treatment trial for Glycogen Storage Disease Type III
Eligibility 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.
Sites / Locations
- Copenhagen Neuromuscular Center, department 3342, RigshospitaletRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
FAXE Kondi - a sugary soft-drink
FAXE Kondi Free - a sugarfree soft-drink
Arm Description
100 ml FAXE Kondi (10 grams of carbohydrates per 100 ml) is ingested every ten minutes during exercise plus 400 ml before exercise start.
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.
Outcomes
Primary Outcome Measures
maximal work capacity
Area Under the Curve (AUC)
Secondary Outcome Measures
Peak oxygen consumption
(VO2peak)
Peak workload
(Wpeak)
Peak respiratory exchange ratio
(RER)
p-lactate
Heart rate
Borg score
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02448667
Brief Title
Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies
Official Title
Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies
Study Type
Interventional
2. Study Status
Record Verification Date
January 2017
Overall Recruitment Status
Unknown status
Study Start Date
January 2015 (undefined)
Primary Completion Date
November 2017 (Anticipated)
Study Completion Date
February 2018 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Rigshospitalet, Denmark
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease Type III
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
FAXE Kondi - a sugary soft-drink
Arm Type
Experimental
Arm Description
100 ml FAXE Kondi (10 grams of carbohydrates per 100 ml) is ingested every ten minutes during exercise plus 400 ml before exercise start.
Arm Title
FAXE Kondi Free - a sugarfree soft-drink
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Dietary Supplement
Intervention Name(s)
FAXE Kondi
Intervention Description
Sucrose and glucose containing softdrink
Intervention Type
Dietary Supplement
Intervention Name(s)
Faxe Kondi Free
Intervention Description
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 Measure Information:
Title
maximal work capacity
Description
Area Under the Curve (AUC)
Time Frame
After up to 1 hour of bicycling on the 2nd and 4th day.
Secondary Outcome Measure Information:
Title
Peak oxygen consumption
Description
(VO2peak)
Time Frame
After up to 1 hour of cycling on the 2nd and 4th day.
Title
Peak workload
Description
(Wpeak)
Time Frame
After up to 1 hour of cycling on the 2nd and 4th day.
Title
Peak respiratory exchange ratio
Description
(RER)
Time Frame
After up to 1 hour of cycling on the 2nd and 4th day.
Title
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.
Title
Heart rate
Time Frame
Continously during the cycle test (max. 1 hour) on the 2nd and 4th day
Title
Borg score
Time Frame
Measured periodically during the cycle test (max. 1 hour) on the 2nd and 4th day
Other Pre-specified Outcome Measures:
Title
Respiratory exchange ratio, RER
Time Frame
measured continously during the exercise test day 2 and 4.
Title
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.
Title
Pain
Description
Visual analog scale (VAS)
Time Frame
Assessed on days 3 and 5 of the trial
Title
Fatigue
Description
Fatigue Severity Score (FSS)
Time Frame
Assessed on days 3 and 5 of the trial
Title
p-Creatine kinase
Description
To asses muscle damage
Time Frame
measured on day 1, 3 and 5.
Title
p-myoglobin
Description
To asses muscle damage
Time Frame
measured on day 1, 3 and 5.
Title
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.
Title
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.
Title
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.
Title
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.
Title
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.
Title
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.
Title
p-sodium
Description
To eliminate electrolyt status as a confounding factor
Time Frame
before exercise on day 2 and 4
Title
p-potassium
Description
To eliminate electrolyt status as a confounding factor
Time Frame
before exercise on day 2 and 4
Title
Hypoglycemic episodes
Time Frame
2 hour observation after each of the two exercise test.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Astrid E Buch, BSc Medicine
Phone
+45 35 45 61 35
Email
astrid.emilie.buch.02@regionh.dk
First Name & Middle Initial & Last Name or Official Title & Degree
Nicolai Preisler, MD
Phone
+45 35 45 61 26
Email
nicolai.preisler@regionh.dk
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Astrid E Buch, BSc Medicine
Organizational Affiliation
Copenhagen Neuromuscular Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Copenhagen Neuromuscular Center, department 3342, Rigshospitalet
City
Copenhagen
State/Province
Region hovedstaden
ZIP/Postal Code
2100
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Astrid E Buch, MD
Phone
+45 35 45 61 35
Email
astrid.emilie.buch.02@regionh.dk
First Name & Middle Initial & Last Name & Degree
Nicolai Preisler, MD
Phone
+45 35 45 61 26
Email
nicolai.preisler@regionh.dk
12. IPD Sharing Statement
Plan to Share IPD
No
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Links:
URL
http://ec.europa.eu/food/fs/sc/scf/out52_en.pdf
Description
Scientific Committee on Food. Re-evaluation of acesulfame K with reference to the previous SCF opinion of 1991.
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Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies
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