UX007-CL202

• Research type

  Research Study

• Full title

  An Open Label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies

• IRAS ID

  205738

• Contact name

  Stephanie Grunewald

• Contact email

  stephanie.grunewald@gosh.nhs.uk

• Sponsor organisation

  Ultragenyx Pharmaceutical Inc.

• Eudract number

  2016-000322-19

• Clinicaltrials.gov Identifier

  NCT02214160

• Duration of Study in the UK

  3 years, 0 months, 1 days

• Research summary

  Research Summary
  The study is an interventional, open-label, long-term safety and efficacy extension study of UX007 or triheptanoin treatment in approximately 100 LC-FAOD subjects who have participated in prior clinical studies or treatment programs with UX007/triheptanoin, or who have failed conventional therapy and have documented severe unmet need (in the opinion of the investigator and sponsor). UX007 dosing will be targeted at 25-35% of total caloric intake. If a subject has been receiving a lower dose of UX007/triheptanoin, the individual
  may continue treatment at the current dose. If however, the subject has symptomatic disease
  or clinical signs of exercise limitation while on UX007 doses below the target range, the investigator should consider titrating the dose to 25-35% of total caloric intake. If a subject has been off UX007/triheptanoin treatment for > 1 month or is treatment naïve, the dose will be titrated following dose administration guidelines. The subjects will be followed to evaluate the long-term safety and continued effects of UX007 for approximately 3 years or until market approval.

  Summary of Research
  A PDF of the lay summary can be found on https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.ultragenyx.com%2Fclinical-trials%2F&data=04%7C01%7Capprovals%40hra.nhs.uk%7C7ad12783aa3a423725fa08d98f7e9d4c%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C637698596233243330%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C1000&sdata=gJBpYEMMwJqelnic3UG3DCv6jVma3GBf%2FmXbJXfFGgE%3D&reserved=0 within 1 year of the global end of trial.

• REC name

  North East - Newcastle & North Tyneside 2 Research Ethics Committee

• REC reference

  16/NE/0170

• Date of REC Opinion

  6 Jun 2016

• REC opinion

  Further Information Favourable Opinion