Eltrombopag in children and young adults with aplastic anemia

• Research type

  Research Study

• Full title

  A phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naive severe aplastic anemia or recurrent aplastic anemia

• IRAS ID

  228843

• Contact name

  Phillip Ross

• Contact email

  philip.ross@novartis.com

• Sponsor organisation

  Novartis Pharmaceuticals UK Limited

• Eudract number

  2015-003166-91

• Clinicaltrials.gov Identifier

  NCT03025698

• Duration of Study in the UK

  6 years, 6 months, 0 days

• Research summary

  This study will examine the use of eltrombopag in children (1 to 18 years) who have been diagnosed with severe aplastic anaemia (SAA). SAA is a rare (approximately two in one million children), life-threatening bone marrow failure disease, treatments for which include intensive immunosuppressive therapy or stem cell transplantation. Patients recruited to this study will lack a suitable matched donor or are not candidates for stem cell transplantation. They may or may not have received prior treatment with immunosuppressive therapy.

  The key purpose of the study is to determine how eltrombopag is processed by the body ('pharmacokinetics') when given to these patients, as well as looking at its safety profile and effect on the patients SAA.

  Patients will be divided into two groups: depending broadly speaking these are a group who have received no treatment with immunosuppressive therapy for their SAA and those who have.
  Patients will begin treatment with a fixed dose of eltrombopag, derived from studies conducted in adults. The dose will differ dependant upon whether the patient falls into either of two age-brackets: one to up to six years and six years to up to eighteen years. Dose will be modified during treatment depending on blood results and protocol guidelines. Eltrombopag will be administered for 26 weeks, follow up for 52 weeks and long term follow up for 3 years. Patients who, in the opinion of the Investigator, are deemed to still be deriving clinical benefit from treatment after the twenty-six week treatment period may be permitted to continue treatment in the follow-up periods.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  17/EM/0239

• Date of REC Opinion

  22 Aug 2017

• REC opinion

  Further Information Favourable Opinion