CTL019 in first-line high-risk pediatric & young adult B-ALL patients

• Research type

  Research Study

• Full title

  A phase II trial of tisagenlecleucel in first-line high-risk (HR) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who are minimal residual disease (MRD) positive at the end of consolidation (EOC) therapy

• IRAS ID

  251668

• Contact name

  Ajay Vora

• Contact email

  Ajay.Vora@gosh.nhs.uk

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2017-002116-14

• Duration of Study in the UK

  7 years, 11 months, 10 days

• Research summary

  A study to find out if the investigational therapy tisagenlecleucel is safe and has beneficial effects in children and young adults who have high-risk B-cell Acute Lymphoblastic Leukaemia (B-ALL).

  The purpose is to test an experimental approach called gene transfer in patients considered to have high-risk ALL. Patients will have received induction and consolidation therapy but still have residual disease after completing consolidation therapy. It is expected patients will be on interim maintenance therapy.

  T cells (type of white blood cell (WBC)) will be removed from the blood and changed in a way that allows them to identify and kill tumour cells. The modification is done by gene transfer and results in a genetic change to the T cells. This allows the changed T cells to recognise B-cells (type of WBC) including tumour cells. These changed cells are tisagenlecleucel.

  T cells collected from a patient after induction therapy or consolidation therapy will be used. Therefore, patients do not need to repeat the leukapheresis procedure unless deemed necessary.

  Benefits of tisagenlecleucel are not established. Treatment may help keep disease in remission or there may be no benefit.

  Patients will receive one dose of tisagenlecleucel by intravenous infusion. Patients may receive a second dose.
  
  Participation will last no more than 8 years.

  The study is made up of the following visits:
  Screening
  Leukapheresis
  Enrolment
  Pre-treatment (lymphodepleting chemotherapy & pre-infusion)
  Tisagenlecleucel infusion & second infusion (if applicable)
  Post-tisagenlecleucel infusion visits
  End of treatment & follow-up
  Relapse & survival follow-up

  Patients will visit the hospital several times before infusion.
  After infusion, patients will be hospitalised for at least ten days and visit:
  Every few days for the first month
  Every 3 months for the first year
  At Months 18 and 24
  Then every 12 months until the study ends.

  The study is organised and funded by Novartis Pharma AG and run by medical staff in hospitals.

• REC name

  South Central - Oxford A Research Ethics Committee

• REC reference

  18/SC/0625

• Date of REC Opinion

  13 Mar 2019

• REC opinion

  Further Information Favourable Opinion