Phase II paediatric study of ostilodrostat in Cushing's patients

• Research type

  Research Study

• Full title

  A phase II, multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing’s disease

• IRAS ID

  253366

• Contact name

  Helen Storr

• Contact email

  h.l.storr@qmul.ac.uk

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2018-001522-25

• Duration of Study in the UK

  3 years, 0 months, 22 days

• Research summary

  This is a research study of a drug called osilodrostat. It is hoped that this drug will be helpful in the treatment of children and young adults with Cushing’s Disease.

  The purpose of the study is to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescents 6 to <18 years of age with Cushing’s disease.
  The population of the study are male and female children and adolescents from 6 to < 18 years of age with Cushing’s disease, for whom pituitary surgery is not an option or for whom surgery has failed or who are awaiting surgery.

  Cushing’s Disease (CD) is caused by an ACTH-secreting pituitary corticotroph adenoma is the most common cause of Cushing’s Syndrome (CS) accounting for about 75-80% of cases. The disease is associated with significant morbidity.
  This study uses a medicine that contains the active substance osilodrostat. The study drug is provided as film-coated tablets containing either 1, 5 or 10 mg osilodrostat. Osilodrostat tablets are used to treat Cushing’s disease, a condition in which the body produces too much of a hormone called cortisol. Osilodrostat blocks excessive production of cortisol in the adrenal glands and therefore may improve the symptoms of Cushing’s disease.

  We expect that around 12 children and young adults will be treated across the world, including 2 patients at 2 hospitals in the United Kingdom.

• REC name

  London - Central Research Ethics Committee

• REC reference

  18/LO/1808

• Date of REC Opinion

  12 Dec 2018

• REC opinion

  Further Information Favourable Opinion