Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

ClinicalTrials.gov ID: NCT01993186

Public ClinicalTrials.gov record NCT01993186. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Jul 21, 2026, 10:52 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Randomized, Double-blind, Placebo-controlled, Parallel-group, Study to Assess the Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome

Study identification

NCT ID: NCT01993186
Recruitment status: Completed
Study type: Interventional
Phase: Phase 2
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Enrollment: 36 participants

Conditions and interventions

Conditions

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Interventions

UX007 Drug
Placebo Drug

Drug

Eligibility (public fields only)

Age range: 1 Year to 100 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Feb 27, 2014
Primary completion: Sep 19, 2017
Completion: Sep 19, 2017
Last update posted: Jun 18, 2020

2014 – 2017

United States locations

U.S. sites: 8
U.S. states: 5
U.S. cities: 7

Facility	City	State	ZIP	Site status
Children's Hospital Colorado - University of Colorado, Denver, School of Medicine	Aurora	Colorado	80045	—
Miami Children's Research Institute	Miami	Florida	33155	—
Neurology & Epilepsy Research Center	Orlando	Florida	32819	—
Columbia University - Department of Neurology	New York	New York	10032	—
Columbia University Medical Center	New York	New York	10032	—
Cook Children's Hospital	Fort Worth	Texas	76104	—
University of Texas Neurometabolic Clinic	Houston	Texas	77030	—
Seattle Children's Hospital	Seattle	Washington	98105	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 6 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT01993186, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Jun 18, 2020 · Synced Jul 21, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01993186 live on ClinicalTrials.gov.

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