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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02432768 |
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Date of registration:
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04/11/2014 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)
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Scientific title:
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The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) |
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Date of first enrolment:
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April 2015 |
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Target sample size:
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22 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02432768 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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Denmark
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Contacts
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Name:
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Karen L Madsen, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Neuromuscular Research Unit, Rigshospitalet |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Genetically and/or biochemically verified diagnosis of McArdle disease
- Body Mass Index of 18-32
- Capacity to consent
Exclusion Criteria:
- Significant cardiac and pulmonary disease
- Pregnancy
- Treatment with beta-blockers
- Inability to perform cycling exercise
- Any other significant disorder that may confound the interpretation of the findings
Age minimum:
18 Years
Age maximum:
65 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Glycogen Storage Disease Type V
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Intervention(s)
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Drug: Triheptanoin
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Other: Placebo oil
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Primary Outcome(s)
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Change in heart rate during constant load cycling exercise (HRconst) with Triheptanoin vs. placebo treatment
[Time Frame: Day 14 and day 28]
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Secondary Outcome(s)
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Change in maximal oxidative capacity (VO2max) with Triheptanoin vs. placebo treatment
[Time Frame: Day 14 and day 28]
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Change in self-rated severity of fatigue symptoms with Triheptanoin vs. placebo treatment
[Time Frame: Day 14 and day 28]
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Change in urine concentrations of organic acids with Triheptanoin vs. placebo treatment
[Time Frame: Day 14 and day 28]
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Change in maximal workload capacity (Wmax) with Triheptanoin vs. placebo treatment
[Time Frame: Day 14 and day 28]
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Change in plasma concentrations of metabolites, citric acid cycle (CAC) intermediates with Triheptanoin vs. placebo treatment
[Time Frame: Day 14 and day 28]
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Secondary ID(s)
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2014-003644-12
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H-8-2014-006
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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