Efficacy & Safety of UX007 in Movement Disorders associated to Glut1DS

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 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)

• IRAS ID

  204303

• Contact name

  Kailash Phatechand Bhatia

• Contact email

  k.bhatia@ucl.ac.uk

• Sponsor organisation

  Ultragenyx Pharmaceutical Inc

• Eudract number

  2015-005536-17

• Clinicaltrials.gov Identifier

  NCT02960217

• Clinicaltrials.gov Identifier

  EMA/190573, Unique Product Identifier (UPI)

• Duration of Study in the UK

  3 years, 6 months, 1 days

• Research summary

  This is a phase 3 study to assess the effectiveness and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS).

  (Glut1 DS) is a rare, severely debilitating disease characterised by seizures, developmental delay, and movement disorders. Glut1 DS is caused by a mutation in SLC2A1, encoding the Glut1 protein responsible for transporting glucose across the blood brain barrier. Lack of glucose in this disorder results in a chronic state of cerebral energy deficiency.

  Current management of Glut1 DS consists of the ketogenic diet (KD) which has been shown to be effective in controlling seizures associated with Glut1 DS (Pong et al. 2012, Pearson et al. 2013). UX007 may help the symptoms of this disease.

  The study will enroll approximately 40 pediatric, adolescent, and adult patients who are not on KD and are having disabling paroxysmal movement disorders.

  There is a 6-week Run-in Period, at the end of which, eligible patients will be randomised (1:1 ratio) to one of two treatment sequences (UX007/placebo or placebo/UX007). At Randomisation, patients will begin a 10-week double-blind Treatment Period 1. Treatment Period 1 will consist of a 2-week titration
  period and an 8-week Maintenance Period. At the end of Treatment Period 1, patients will discontinue treatment and begin a 2-week washout period to minimise any potential carryover effect.

  Patients will crossover to the second randomised, double-blind treatment assignment (placebo to UX007, UX007 to placebo) for an additional 10 weeks during Treatment Period 2. Treatment Period 2 will consist of a 2-week titration period and an 8-week Maintenance Period. At the end of the blinded crossover period (Week 22), all active patients will have the option of rolling into the open label Extension Period, to continue UX007 treatment for up to 3 years.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  17/LO/0640

• Date of REC Opinion

  4 Aug 2017

• REC opinion

  Further Information Favourable Opinion