NCT02014883

Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Institut National de la Santé Et de la Recherche Médicale, France · Other Government
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this project is to study the efficacy of triheptanoin oil in patients with GLUT1 deficiency syndrome.

Detailed description

The primary objective of the study is: \- to evaluate the capacity of triheptanoïn to improve the condition of patients with GLUT1-DS The secondary objectives of the study are: * to confirm the short-term safety of triheptanoïn therapy in patients with GLUT1-DS * to evaluate the short-term effects of triheptanoïn treatment on motor function, autonomy, quality of life and clinical signs of patients with GLUT1-DS * to evaluate the effect of triheptanoïn on brain energy metabolism using non-invasive 31P-MRS spectroscopy after activation of the occipital cortex in order to measure the levels of high-energy phosphates (such as ATP and phosphocreatine)

Conditions

• Glut1 Deficiency Syndrome

Interventions

Type	Name	Description
DRUG	GLUT1 DS

Timeline

• Start date: 2013-12-04
• Primary completion: 2019-07-04
• Completion: 2019-07-04
• First posted: 2013-12-18
• Last updated: 2025-10-03

Locations

1 site across 1 country: France