NCT02968953

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

Treatment With UX007 (Triheptanoin) for a Single Patient (ERS) With Glucose Transporter 1 (GLUT1) Deficiency Syndrome

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Jerry Vockley, MD, PhD · Academic / Other
• Sex: Female
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome.

Detailed description

This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome. Patient has already tried the other acceptable treatments for GLUT1 with either side effects that caused treatment to be discontinued (Ketogenic Diet) or a current treatment that is not showing any efficacy and is difficult to sustain for a long duration given this patient's age (modified Atkins diet). Due to the patient's ongoing neuroregression and lack of other viable treatments, the use of UX007 (triheptanoin) is warranted. There is no comparable or satisfactory alternative therapy to treat this patient's severely debilitating condition and the potential benefits to the patient justify the potential risks of the treatment. The potential risks from UX007 (triheptanoin) are not unreasonable given the context of this patient's condition.

Conditions

• Glucose Transporter 1 Deficiency Syndrome

Interventions

Type	Name	Description
DRUG	Triheptanoin	A single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome will receive tripheptanoin (UX007).

Timeline

• First posted: 2016-11-21
• Last updated: 2019-02-08

Locations

1 site across 1 country: United States