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Triheptanoin in Mc Ardle

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ClinicalTrials.gov Identifier: NCT02919631
Recruitment Status : Unknown
Verified July 2016 by Institut National de la Santé Et de la Recherche Médicale, France.
Recruitment status was:  Not yet recruiting
First Posted : September 29, 2016
Last Update Posted : September 29, 2016
Sponsor:
Collaborator:
Rigshospitalet, Denmark
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

Brief Summary:

Background: Patients with the sugar metabolism disorder, Glycogen Storage Disease Type V, have insufficient breakdown of sugar stored as, glycogen, within the cells. The investigators know from previous studies with McArdle patients, that they not only have a reduced sugar metabolism, both also have problems in increasing their fat metabolism during exercise to fully compensate for the energy deficiency.

Studies on Triheptanoin diet used in patients with other metabolic diseases have shown that Triheptanoin can increase metabolism of both fat and sugar. In these patients, Triheptanoin has had a positive effect on the physical performance and has reduces the level of symptoms experienced by patients.

Aim: To investigate the effect of treatment with the dietary oil, Triheptanoin, in patients with McArdle disease on exercise capacity.

Methods: 20-30 adult patients will be recruited through Rigshospitalet in Copenhagen, Denmark and Hopital Pitié-Sapêtrière in Paris, France.

  1. Pre-experimental testing (1 day): Baseline blood samples are collected to obtain baseline values of safety parameters: Plasma-acylcarnitines, free fatty acids and creatine kinase. Subjects perform a max-test to determine their VO2max
  2. Treatment period #1 (2 weeks): Subjects follow a diet consuming a dietary treatment oil. Neither patients nor members of the study group know who receive which type of oil.
  3. Washout period (1 week +7 days): Subjects receive no treatment
  4. Treatment period #2 (2 weeks +7days): Subjects who received Triheptanoin oil in the first treatment period, now receive placebo oil and vice versa.

Assessments: Before and after each treatment periods, subjects perform a 30-minutes exercise test on a cycle ergometer, comprising of 20-22 minutes of constant load exercise and 6-8 minutes increasing load to peak. Subjects will complete a Fatigue Severity Scale questionnaire and metabolic products will be measured in blood and urine.


Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type V Drug: Triheptanoin Drug: Placebo oil Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: The Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V)
Study Start Date : October 2016
Estimated Primary Completion Date : September 2017


Arm Intervention/treatment
Active Comparator: triheptanoin
14 days on Triheptanoin treatment including a 7 days titration period and a 7 days full dose treatment of 1mL/kg/day.
Drug: Triheptanoin
Anaplerotic dietary oil
Other Name: UX007

Placebo Comparator: placebo oil
14 days of diet on a placebo oil including 7 days titration period and 7 days full dose treatment of 1mL/kg/day
Drug: Placebo oil
Safflower oil
Other Name: UX007 Placebo Oral Liquid




Primary Outcome Measures :
  1. Change in heart rate during constant load cycling exercise (HRconst) with Triheptanoin vs. placebo treatment. [ Time Frame: Day 14 and Day 28 ]
    Subject heart rate will be measured during 20 minutes exercise test performed on a cycle ergometer at a workload corresponding to approximately 60% of maximal oxidative capacity (VO2max).


Secondary Outcome Measures :
  1. Change in maximal oxidative capacity (VO2max) with Triheptanoin vs. placebo treatment. [ Time Frame: Day 14 and Day 28 ]
    The maximal oxidative capacity measured at peak workload after a 6 minutes ramp test performed on a cycle ergometer after 20 minutes constant load cycling.

  2. Change in self-rated severity of fatigue symptoms with Triheptanoin vs. placebo treatment. [ Time Frame: Day 14 and 28 ]
    Total score on a Fatigue Severity Scale (FSS)

  3. Change in urine concentrations of organic acids with Triheptanoin vs. placebo treatment. Urine concentrations of: 3OH-propionate, heptanoate, methylmalonate, pimelate methylcitrate [ Time Frame: Day 14 and 28 ]
  4. Change in maximal workload capacity (Wmax) with Triheptanoin vs. placebo treatment. [ Time Frame: Day 14 and Day 28 ]
    The maximal workload capacity (Wmax) is measured at peak workload after a 6 minutes ramp test performed on a cycle ergometer after 20 minutes constant load cycling.

  5. Change in plasma concentrations of metabolites, citric acid cycle (CAC) intermediates with Triheptanoin vs. placebo treatment. [ Time Frame: Day 14 and Day 28 ]
    Plasma concentrations of metabolites and citric acid cycle (CAC) intermediates: Lactate, ammonia, glucose, Free Fatty Acids (FFA), acyl-carnitines and malate (a CAC intermediate).



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female between 18 and 65 years of age,
  • Genetically and/or biochemically verified diagnosis of McArdle disease
  • Body Mass Index of 18-32
  • Women in child-bearing age on contraceptive treatment with: Birth control pills, coil, ring, transdermal hormone patch injection of gestagen or subdermal implant.
  • French national health insurance
  • Signed informed consent
  • Available for phone calls

Exclusion Criteria:

  • Minor
  • Significant cardiac or pulmonary disease
  • Pregnancy (confirmed by urinary-HCG) or breastfeeding. Pregnancy not planned and to be avoided during the study by use of effective contraceptive methods.
  • Persons deprived of their liberty by judicial or administrative decision
  • Adult subject under legal protection or unable to consent
  • Treatment with beta-blockers
  • Inability to perform cycling exercise
  • Any other significant disorder that may confound the interpretation of the findings
  • Person subject to an exclusion period for a previous clinical trial

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): NCT02919631


Contacts
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Contact: Pascal LAFORET, PH + 33 (0)1 42 16 37 76 pascal.laforet@psl.aphp.fr

Sponsors and Collaborators
Institut National de la Santé Et de la Recherche Médicale, France
Rigshospitalet, Denmark

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Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT02919631     History of Changes
Other Study ID Numbers: C15-54'
First Posted: September 29, 2016    Key Record Dates
Last Update Posted: September 29, 2016
Last Verified: July 2016
Additional relevant MeSH terms:
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Glycogen Storage Disease
Glycogen Storage Disease Type V
Metabolic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn