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Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

0
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
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0
0
0
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Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Ultragenyx Pharmaceutical
2 programs
UX007PHASE_21 trial
UX007PHASE_31 trial
Active Trials
NCT01993186Completed36Est. Sep 2017
NCT02960217Terminated44Est. Oct 2019

Trial Timeline

Clinical trial activity over time

2021
2022
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2024
2025
2026
Ultragenyx PharmaceuticalUX007
Ultragenyx PharmaceuticalUX007

Clinical Trials (2)

Total enrollment: 80 patients across 2 trials

NCT02960217Ultragenyx PharmaceuticalUX007

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Start: Apr 2017Est. completion: Oct 201944 patients
Phase 3Terminated
NCT01993186Ultragenyx PharmaceuticalUX007

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

Start: Feb 2014Est. completion: Sep 201736 patients
Phase 2Completed

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

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.