Paediatric venetoclax phase I study
Research type
Research Study
Full title
A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Paediatric and Young Adult Patients with Relapsed or Refractory Malignancies
IRAS ID
237080
Contact name
Kate Honeybill
Contact email
Sponsor organisation
AbbVie Ltd
Eudract number
2017-000439-14
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 11 months, 7 days
Research summary
Paediatric patients with relapsed (cancer has come back) or refractory (does not respond to cancer treatments) cancers have poor prognosis. Venetoclax is a drug that blocks a protein in cells called B-cell lymphoma 2 or BCL-2. When there is extra BCL-2 in cancer cells, it can help the cancer cells to stay alive.
Venetoclax is an experimental drug that kills cancer cells by blocking a protein (part of a cell) that allows cancer cells to stay alive. This study will look at how venetoclax works on its own and how it works with approved chemotherapy drugs. This is the first study of venetoclax in children and young adults.
This is a Phase 1, open-label study in paediatric and young adult cancer patients who have previously received treatment for their disease. All patients in the study will receive venetoclax, administered orally once daily (QD) with or without chemotherapy at the discretion of the study doctor.
The study consists of two parts: Part 1 and Part 2.
In Part 1 patients with any relapsed or refractory cancer may enrol. Part 1 will determine the dose of venetoclax. This dose will also be used in Part 2.
During Part 2 patients with the following malignancies: acute lymphoblastic leukaemia (ALL), acute myeloid leukaemia (AML), non-Hodgkin lymphoma (NHL), or neuroblastoma are eligible to enrol. In addition patients with any tumour that expresses BCL-2 can also enrol.
About 165 patients will take part in this study at about 40 sites globally.
Patients will continue to have study visits and receive treatment for as long as they are having a clinical benefit. The effect of the treatment on the cancer will be checked by blood/cancer assessment tests and checking for side effects.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
19/NE/0130
Date of REC Opinion
22 Jul 2019
REC opinion
Further Information Favourable Opinion