The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses

• Register: EUCTR
• Last refreshed on: 21 September 2020
• Main ID: EUCTR2017-004153-17-DK
• Date of registration: 26/03/2018
• Prospective Registration: Yes
• Primary sponsor: Copenhagen Neuromuscular Center
• Public title: The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses
• Scientific title: Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses
• Date of first enrolment: 03/05/2018
• Target sample size: 20
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004153-17
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: yes; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Denmark

Contacts

• Name: Copenhagen Neuromuscular Center
• Affiliation: Rigshospitalet

• Name: Copenhagen Neuromuscular Center
• Affiliation: Rigshospitalet

Key inclusion & exclusion criteria

Inclusion criteria:
- Male/Female Age > 15 years
- Genetically and/or biochemically verified diagnosis of GSD III, GSD VII or GSD XV
- Capacity to consent
- For women in fertile age on contraceptive treatment with: Birth control
pills, coil, ring, transdermal hormone patch injection of gestagen or
subdermal implant.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 14
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion criteria:
- Significant cardiac or pulmonary disease
- Pregnancy (confirmed by urine stix or plasma-HCG) or breastfeeding.
- Inability to perform cycling exercise
- Any other significant disorder that may confound the interpretation of
the findings


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency. Tarui's disease Also called: glycogen storage disease Type VII or phosphofructokinase deficiency. Glycogenin-1 deficiency or glycogen storage disease Type XV.
MedDRA version: 20.1 Level: PT Classification code 10053241 Term: Glycogen storage disease type VII System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1 Level: PT Classification code 10053250 Term: Glycogen storage disease type III System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: LLT Classification code 10053255 Term: Tarui disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: LLT Classification code 10016983 Term: Forbes' disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Triheptanoin
Product Code: UX007
Pharmaceutical Form: Oral liquid
INN or Proposed INN: Not available
CAS Number: 210-647-2
Current Sponsor code: UX0007
Other descriptive name: TRIHEPTANOIN
Concentration unit: % percent
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral liquid
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: To investigate the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise.

Secondary Objective: To investigate the effect of Triheptanoin treatment on:
* Self-rated daily function scores on a modified SF-36 questionnaire
* Maximal workload capacity (Wmax).
* Changes in plasma concentrations of lactate, ammonia, glucose, FFA, acyl-carnitines, malate (a TCA intermediate), C5, and hormones.
* Rate of Perceived Exertion (Borg score) during constant workload cycling.
* Maximal oxidative capacity.
* Bouchards energy expenditure questionnaire.
* Glucose rate of appearance and disappearance.

Primary end point(s): - Heart rate during constant load cycling exercise (HR_const)
- Fatty acid oxidation (FAO)

Timepoint(s) of evaluation of this end point: Before and after 14 days of treatment with Triheptanoin and before and
after 14 days of placebo treatment.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Before and after 14 days of treatment with Triheptanoin and before and after 14 days of placebo treatment.

Secondary end point(s): • Self-reported health and well-being addressed in an SF-36 Questionnaire.
• Maximal workload capacity (Wmax).
• Plasma concentrations of lactate, ammonia, glucose, FFA, acyl-carnitines and malate (a TCA intermediate), creatine kinase, and hormones.
• Rate of Perceived Exertion during constant workload cycling (RPEconst).
• Total fatty acid oxidation and total carbohydrate oxidation measured by indirect calorimetry.
• Glucose rate of appearance and disappearance.

Secondary ID(s)

20171012

Source(s) of Monetary Support

Novo Nordisk Foundation

A.P. Møller Foundation

Ultragenyx Pharmaceuticals Inc.

Ethics review

Status: Approved
Approval date: 03/05/2018
Contact: