New Clinical Trial Announced for Patients with Glucose Transporter Type-1 Deficiency Syndrome

March 28, 2017

For Patients With Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS) Experiencing Movement Disorders Learn about a Phase 3 clinical research

A study in Glut1 DS

What Is Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)?

Glut1 DS is caused by a genetic defect in the transport of glucose into the brain.1

Symptoms generally fall into 3 categories:

• Seizures
• Developmental delays
• Movement disorders

(Multiple types of movement disorders can occur due to Glut1 DS, including paroxysmal dyskinesias (pa-rox-is-mal dis-kin-ee-see-as)

• These are movement disorders that are episodic, which means they only happen at certain times

° They may happen with or without seizures


A Phase 3 clinical study in people with Glut1 DS is now enrolling.

You may be eligible to enroll if you meet the following key criteria:

  • Diagnosis of Glut1 DS
  • Age 6 years or older
  • Experience movement disorder events that impact or limit physical functioning and/or activities of daily living at least once per month° Examples of ways in which movement disorder events might impact or limit activities include caring for oneself, walking, attending or participating in school or work, or doing sports or exercise

    • Not on, or not regularly following, a special diet, such as the ketogenic diet, for 3 months prior to screening

    ° People who are doing well and who are stable on the ketogenic diet should not discontinue the diet in order to participate in this study.

    What should I know about the Phase 3 clinical study?

  • The study will collect information on safety and efficacy of UX007 in people with Glut1 DS with paroxysmal movement disorders
  • The study is 28 weeks long, and is a randomized, double-blind, placebo-controlled crossover study. This means that:
    • °  At the beginning of the study, half of all subjects will receive UX007, and half will receive a placebo (looks like the study drug but does not contain the study drug’s active ingredient)
    • °  At the end of the first part of the study, participants who received UX007 will be switched to placebo, and those who received placebo will be switched to UX007• After the study, participants may have the opportunity to take part in an extension study in which they will receive UX007


For more information:

Visit (search using NCT02960217)

2. Visit

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