D5136C00007 Ticagrelor for sickle cell disease in paediatric patients

• Research type

  Research Study

• Full title

  Multicenter, open-label, randomised, pharmacokinetic (PK) and pharmacodynamic (PD) dose-ranging Phase II study of ticagrelor followed by a double-blind, randomised, parallel-group, placebo-controlled 4 weeks extension phase in paediatric patients with sickle cell disease

• IRAS ID

  159417

• Contact name

  Philip Connor

• Contact email

  philip.connor@wales.nhs.uk

• Sponsor organisation

  AstraZeneca AB

• Eudract number

  2014-001006-18

• Research summary

  This is a phase 2 dose-ranging paediatric study of ticagrelor to investigate the relationship between the dose of ticagrelor and the inhibition of platelet aggregation in children with sickle cell disease.
  A minimum of 36 participants and a maximum of 50 participants will be randomised in the study. The study will be conducted in 4 countries (USA, UK, South Africa and Canada) and will be divided into 2 parts (Part A and B).
  Part A: Suitable participants will be randomised to receive one of 2 dosing schedules. All patients will receive 0.125 mg/kg of ticagrelor as their initial dose. This will be followed by a dose of either 0.375 mg/kg or 0.563 mg/kg 7 days later.
  Part B:
  These patients will then be randomised (2:1 ratio) to ticagrelor 0.125 mg/kg twice daily or placebo for a 4-week treatment phase. This part of the study will be double blinded which means that neither the participants nor the research team will know which treatment the participants are on.

  Participants will be expected to be in the study for approximately 3 months during which time they will attend 9 clinic visits. The following are some of the study procedures that will be performed during study visits: blood tests, urine tests,
  electrocardiograms and physical examination. Participants will also be provided with an e-diary which they will be expected to complete daily before bedtime.

  It is hoped the results of this study will help to support the dose selection for the phase 3 study and ultimately for the development of an effective alternative treatment option for patients with sickle cell disease.

• REC name

  East Midlands - Leicester South Research Ethics Committee

• REC reference

  14/EM/1071

• Date of REC Opinion

  27 Aug 2014

• REC opinion

  Further Information Favourable Opinion