Ponatinib in Recurrent/Refractory Pediatric Leukemias & solid Tumors
Research type
Research Study
Full title
An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias or Solid Tumors in Pediatric Participants
IRAS ID
264830
Contact name
Lynley Marshall
Contact email
Sponsor organisation
Incyte Biosciences International Sàrl
Eudract number
2018-004878-99
Duration of Study in the UK
3 years, 5 months, 1 days
Research summary
This is a phase I/2 open label study to evaluate the safety and anti-tumour activity of Ponatinib in paediatric and adolescent participants with malignancies (cancer) who are refractory or relapsed from prior standard therapy or not eligible for further standard therapy. Leukaemia will be the primary disease of relevance to Ponatinib though patients with other relevant tumour types will be included.
Ponatinib’s class is a Tyrosine Kinase Inhibitor (TKI). TKIs target two chromosomes in which have 'broken' and joined up abnormally in the leukaemia cells, called BCR-ABL or the Philadelphia chromosome. Ponatinib is a new oral medication which can currently be given to adults with CML. Although CML is rare in children there are not a lot of options for children who are resistant or intolerant to the current standard treatment.
The study will evaluate first the Maximum tolerated dose and how safe and tolerated Ponatinib is. There will be 18-24 participants split into age categories of 12-18, 6-12- and 1-6-year olds. The study will then expand to evaluate efficacy using a dose calculated from phase 1. There will be up to 36 participants in this expansion.
There will be three periods of the study, screening (28 days), daily treatment (28-day cycles) and follow-up (30-37 days after last dose). Number of cycles for each participant are based on benefit and having not met criteria for withdraw so participation will vary but is expected to be 6-24 months. Efficacy assessments will be after every 2 cycles from the outset, and patients will be withdrawn from study if they have disease progression or are not tolerating the drug.Safety will be tested throughout and will include bloods, ECG, disease-relevant scans, physical examinations. Study assessments may differ dependent on tumour type.
Incyte Biosciences international Sàrl is the sponsor for this study.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
20/EM/0082
Date of REC Opinion
22 Jun 2020
REC opinion
Further Information Favourable Opinion