The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses
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|ClinicalTrials.gov Identifier: NCT03642860|
Recruitment Status : Recruiting
First Posted : August 22, 2018
Last Update Posted : October 2, 2018
The aim of this study is to investigate the effect of 14 days of treatment with the dietary oil-supplement Triheptanoin on fat metabolism and exercise tolerance in patients with Phosphofructokinase deficiency, Debrancher deficiency and Glycogenin-1 deficiency. The investigators wish to investigate whether a Triheptanoin diet can improve exercise capacity by measuring:
- Heart rate during cycling exercise and maximal exercise capacity
- Fat and glucose metabolism
- Concentrations of metabolic substrates in blood during exercise
- Perception of fatigue and symptoms by questionnaire
- Degree of exhaustion during cycling exercise by Borg score
All measurements are done before and after 14 days with a Triheptanoin-oil diet, and before and after 14 days diet with safflower (Placebo-oil).
Triheptanoin-oil supplementation in the diet has been shown to increase metabolism of both fat and carbohydrates in patients with other metabolic myopathies. In these patients, Triheptanoin improved physical performance and has reduced the amount of symptoms experienced by patients.
|Condition or disease||Intervention/treatment||Phase|
|Tarui Disease Debrancher Deficiency GYG1 DEFICIENCY||Drug: Triheptanoin Drug: Placebo Oil||Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Crossover Assignment|
|Intervention Model Description:||The study will be conducted as a randomized, placebo-controlled, double blind, crossover study consisting of two 14 days treatment periods set apart by minimum a 7-days wash-out period where no treatment is taken. In one treatment period, the subjects receive UX007 treatment and placebo treatment in the other. The study will consist of three separate groups; one group consisting of GSD III, one group of GSD VII, and one group of GSD XV subjects. Each of these groups will be blinded and randomized separately|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Triheptanoin's Effect on Fatty Acid Oxidation and Exercise Tolerance in Patients With Debrancher Deficiency, Glycogenin-1 Deficiency and Phosphofructoinase Deficiency at Rest and During Exercise. A Randomized, Double-blind, Placebo-controlled, Cross-over Study|
|Actual Study Start Date :||August 15, 2018|
|Estimated Primary Completion Date :||October 31, 2019|
|Estimated Study Completion Date :||December 31, 2019|
Experimental: Active treatment
Daily treatment with Triheptanoin oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day).
Other Name: Trioctanoin
Placebo Comparator: Placebo treatment
Drug: Placebo Oil
Daily treatment with Safflower oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day).
- Heart rate [ Time Frame: 60 min ]Heart rate during constant load cycling exercise.
- Palmitate oxidation [ Time Frame: 60 min ]Palmitate oxidation measured via stable isotope technique and indiret calorimetry during constant load exercise.
- Short Form-36 Questionnaire [ Time Frame: 2 weeks ]The Short form-36 assesses eight health concepts: limitations in Quality of life physical activities because of health problems; limitations in social activities because of physical or emotional problems; limitations in usual role activities because of physical health problems); bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities because of emotional problems; vitality (energy and fatigue); and general health perceptions. The standard form of the instruments asks for participants to reply to questions according to how they have felt over the previous week. The items use Likert-type scales, from 1-5 points, where 1 usually indicates that the patient is feeling worse.
- Maximal workload capacity [ Time Frame: 60 min ]During cycle exercise
- Plasma concentrations of lactate, ammonia, glucose, FFA, acyl-carnitines, malate, C5, insulin, adrenalin and noradrenalin. [ Time Frame: 60 min ]
- Rate of Perceived Exertion (RPE) [ Time Frame: 60 min ]Borg Score during constant workload cycling. The Borg RPE scale is a numerical scale that ranges from 6 to 20, where 6 means "no exertion at all" and 20 means "maximal exertion. The Borg scale is named after Borg GA 1982.
- Bouchards energy expenditure questionnaire [ Time Frame: 3 days ]Bouchard's Physical Activity Record (BAR) is a widely used diary in which participants report physical activity for each 15 minute interval over three days. Activities are rated on a scale of 1 to 9 (1 = sedentary activity, 9 = intense manual work or high intensity sports) to yield a total energy expenditure score.
- Glucose rate of appearance and disappearance [ Time Frame: 60 min ]
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): NCT03642860
|Contact: Mette Cathrine Ørngreen, MD, DMscfirstname.lastname@example.org|
|Contact: Daniel Emil Raaschou-Pedersen, Medical Studentemail@example.com|
|Copenhagen Neuromuscular Center||Recruiting|
|Copenhagen, Denmark, 2200|
|Contact: Mette Cathrine Ørngreen, MD, DMsc 004535457614 firstname.lastname@example.org|
|Contact: Daniel Emil Raaschou-Pedersen, Medical Student 004535456135 email@example.com|
|Principal Investigator: Mette Cathrine Ørngreen, MD, DMsc|