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

  Masoud Mokhtarani

• Contact email

  Masoud.Mokhtarani@@horizonpharma.com

• Sponsor organisation

  Horizon Therapeutics, Inc.

• Eudract number

  2015-000075-27

• Clinicaltrials.gov Identifier

  073480, IND Number

• Duration of Study in the UK

  2 years, 3 months, 31 days

• Research summary

  This is a Randomised, Controlled, Open-Label Crossover Study that plans to investigate the efficacy, tolerability and safety of RAVICTI as compared with NaPBA
  treatment in patients with Urea Cycle Disorders (UCDs). A urea cycle disorder 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 to keep the level of ammonia in the blood at safe levels.
  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.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  15/SW/0206

• Date of REC Opinion

  11 Sep 2015

• REC opinion

  Unfavourable Opinion