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Triheptanoin in Rare Glycogen Storage Diseases

NCT03642860Phase 2

Key facts

Status: Completed
Start: Aug 2018
Primary completion: Aug 2019
Countries: 1
Sponsor: Rigshospitalet
Modality: SLNs, Nanostructured Lipid Carriers, Small Molecules

Drugs: triheptanoin
Conditions: Glycogen storage disease type IIIglycogen storage disease type VII (GSDVII)Glycogenin-1 deficiency

Overview

Phase 2, randomised, double-blind trial in Glycogen storage disease type III, glycogen storage disease type VII (GSDVII) and Glycogenin-1 deficiency comparing Triheptanoin with Placebo (2 arms). Primary endpoints: Heart rate during cycling exercise and Palmitate oxidation. Started Aug 2018; status: completed.

Design

2 arms

Study design: RandomizedDouble-blind

Triheptanoin
Daily treatment with Triheptanoin oil (1g/kg/day)
Placebo
Daily treatment with Safflower oil (1g/kg/day)

Who it enrols

Patients >15 years with verified Debrancher deficiency, Phosphofructokinase deficiency, or Glycogenin 1 deficiency. Excludes pregnant/breastfeeding women, those on beta-blockers, or those unable to perform cycling exercise.

Endpoints

Primary

Heart rate during cycling exercise60 min
Palmitate oxidation60 min

Secondary

Short Form-36 Questionnaire2 weeks
Maximal workload capacity60 min
Plasma concentrations of lactate, ammonia, glucose, FFA, acyl-carnitines, malate, C5, insulin, adrenalin and noradrenalin60 min
Rate of Perceived Exertion60 min
Bouchards energy expenditure questionnaire3 days
Glucose rate of appearance and disappearance60 min

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