Paediatric Study with Blinatumomab in Acute Lymphoblastic Leukaemia

• Research type

  Research Study

• Full title

  An Open-Label, Multi-center, Expanded Access Protocol of Blinatumomab for the Treatment of Pediatric and Adolescent Subjects with Relapsed and/or Refractory B-precursor Acute Lymphoblastic Leukemia (ALL) (Rialto Study)

• IRAS ID

  159980

• Contact name

  Katharine Patrick

• Sponsor organisation

  Amgen Ltd

• Eudract number

  2014-001700-21

• Duration of Study in the UK

  2 years, 10 months, 24 days

• Research summary

  The purpose of this study is to make blinatumomab available to children and adolescents with relapsed/refractory (R/R) Acute Lymphoblastic Leukemia (ALL) of a certain subtype, called B-precursor ALL. ALL is a cancer in which the bone marrow makes too many immature lymphocytes (a type of white blood cells) and the development of normal blood cell t in the marrow is inhibited.

  Blinatumomab is still experimental and not approved by any regulatory health agency (Food and Drug Administration [FDA] or European Medicines Agency [EMA]) for use in people with R/R ALL.

  The main intent of this study is to provide access to the experimental drug blinatumomab, rather than formally test the study drug. However, data will be collected to see if blinatumomab causes side effects in children and adolescents with R/R ALL and whether it is effective in eliminating tumour cells from the body.

  About 40 children and adolescents are expected to participate in this study. It will take place in approximately 20 centres in the United States, European Union and Switzerland.

  Participants will receive blinatumomab for up to 7.5 months. Blinatumomab will be administered as a continuous intravenous (IV) infusion via a mini pump that can be carried by the participant (in a shoulder- or belt-bag with the pump and infusion bag) for 4 weeks followed by a 2-week treatment-free interval (this is one cycle). Participants can receive up to 5 cycles of blinatumomab. Depending on the participants overall condition, continuous infusion of blinatumomab may be given on an outpatient basis, so study participants may leave the hospital with the pump, if determined to be safe and feasible.

  Participants will be required to attend regular study visits during which they will undergo a number of tests, including: bone marrow aspirate/biopsy, lumbar puncture, blood tests, general and safety assessments.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  15/EM/0066

• Date of REC Opinion

  19 Feb 2015

• REC opinion

  Further Information Favourable Opinion