MedTrace Logo

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

NCT01993186 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2020-06-19

Study results available
· View outcomes & findings →

Summary

The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG).

Conditions

  • Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Interventions

DRUG

UX007

oral liquid

DRUG

Placebo

oral liquid

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-28
Primary Completion
2017-09-20
Completion
2017-09-20

Countries

  • United States
  • Australia
  • France
  • Israel
  • Italy
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01993186 on ClinicalTrials.gov