Olaparib monotherapy in paediatric patients
Research type
Research Study
Full title
A Phase I, Open-label, Parallel Group Study to Investigate Olaparib Safety and Tolerability, Efficacy and Pharmacokinetics in Paediatric Patients with Solid Tumours
IRAS ID
264517
Contact name
Martin Elliott
Contact email
Sponsor organisation
AstraZeneca AB
Eudract number
2018-003355-38
Duration of Study in the UK
6 years, 0 months, 3 days
Research summary
AstraZeneca AB is doing this research to determine if olaparib will be safe and effective for the treatment of children and adolescents with relapsed or treatment resistant (refractory) solid cancers with a specific genetic change called Homologous Recombination Repair (HRR) deficiency. Olaparib (Lynparza™) is a PARP (poly [adenosine diphosphate-ribose] polymerase) inhibitor: this means that olaparib stops an enzyme found in the body known as PARP from working. When deoxyribonucleic acid (DNA; the genetic material of the cell) is damaged in our cells, PARP and the components of the HRR mechanism help to repair the damaged DNA. Olaparib aims to block the repair of DNA which can then cause cancer cells to die. In adults, olaparib has been shown to be effective against cancers with mutations in HRR genes (including BReast CAncer sensitivity genes 1 and 2, BRCA1 and BRCA2). This study will evaluate if this is also true for children and adolescents with cancers with a HRR mutation. Olaparib is not approved for use in children or adolescents by any health authority, except for use in research studies like this. As many as 48 children and adolescents with relapsed or refractory solid cancers from approximately 25 hospitals worldwide may take part in this study; all patients will need to demonstrate HRR deficiency in their tumour or blood. The study will enrol paediatric patients aged from ≥6 months to <18 years. If the patient meets all the criteria to take part in the study, and the parent/guardian and/or patient decide the patient will take part in the study, the patient will be enrolled into one of three age-based treatment groups and assigned to receive olaparib twice daily at a dose appropriate to the patient’s age for as long as the cancer has not worsened (progressed).
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
19/YH/0346
Date of REC Opinion
5 Dec 2019
REC opinion
Further Information Favourable Opinion