Glucose Transporter Type 1 Deficiency Syndrome

2
Pipeline Programs
1
Companies
2
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
2
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Ultragenyx Pharmaceutical
2 programs
2
TriheptanoinPhase 21 trial
UX007Phase 21 trial
Active Trials
NCT02000960Unknown3Est. Oct 2016
NCT02599961Terminated15Est. Oct 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalUX007
Ultragenyx PharmaceuticalTriheptanoin

Clinical Trials (2)

Total enrollment: 18 patients across 2 trials

NCT02599961Ultragenyx PharmaceuticalUX007

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

Start: Sep 2015Est. completion: Oct 201915 patients
Phase 2Terminated
NCT02000960Ultragenyx PharmaceuticalTriheptanoin

Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome

Start: Apr 2014Est. completion: Oct 20163 patients
Phase 2Unknown

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 companies competing in this space