RAVICTI and NaPBA in Patients with Urea Cycle Disorders

• Research type

  Research Study

• Full title

  A Randomised, Controlled, Open-Label Crossover Study of the Safety, Pharmacokinetics and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate [GPB]) Oral Liquid and Sodium Phenylbutyrate (NaPBA) in Phenylbutyrate Treatment Naïve Patients with Urea Cycle Disorders (UCDs)

• IRAS ID

  174139

• Contact name

  Elizabeth Robinson

• Contact email

  brobinson@horizonpharma.com

• Sponsor organisation

  Horizon Therapeutics, Inc.

• Eudract number

  2015-000075-27

• Clinicaltrials.gov Identifier

  073480, IND Number

• Duration of Study in the UK

  1 years, 11 months, 30 days

• Research summary

  This is a randomised, controlled, open­label crossover study that will investigate the safety, pharmacokinetics and ammonia control of RAVICTI as compared with sodium phenylbutyrate (NaPBA) in patients with Urea Cycle Disorders (UCD) who were not previously treated with phenylbutyrate
  UCD is a genetic disorder caused by a deficiency of one of the enzymes in the urea cycle which is responsible for removing ammonia from the blood stream. Medical treatment is aimed at keeping the amount of ammonia in the blood at safe levels. UCD can cause cognitive impairment and other neurological deficits.
  Approximately 30 participants: male and female of 2 years of age and older will be enrolled in 19 centers across different countries.
  The duration of the study is approximately 28 weeks. In addition, there is an an optional 1 year long term safety extension period.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  15/LO/1838

• Date of REC Opinion

  7 Mar 2016

• REC opinion

  Further Information Favourable Opinion