ABT-199 Monotherapy in Relapsed/Refractory CLL With the 17p Deletion
Research type
Research Study
Full title
A Phase 2 Open-Label Study of the Efficacy of ABT-199 (GDC-0199) in Subjects with Relapsed or Refractory Chronic Lymphocytic Leukemia Harboring the 17p Deletion
IRAS ID
117243
Contact name
Peter Hillmen
Contact email
Sponsor organisation
AbbVie Deutschland GmbH & Co. KG
Eudract number
2012-004027-20
Clinicaltrials.gov Identifier
Research summary
Chronic lymphocytic leukaemia (CLL) causes the build-up of immature white blood cells called lymphocytes. Up to 30% of all relapsed/refractory patients with CLL have a missing part in their chromosome 17, labelled 17p deletion. CLL patients with the 17p deletion often do not respond to the current treatments for CLL. There are currently no treatments approved specifically to meet the need of the 17p deletion population.
High amounts of a protein called Bcl-2 are present in patients with leukaemia. These proteins are associated with tumour development, disease progression and drug resistance. ABT-199 is a unique drug, taken orally that inhibits Bcl-2. Laboratory and animal studies have shown that ABT-199 kills cancer cells and cause tumours to shrink. ABT-199 was equally effective against the subset of patient-derived CLL cells bearing the high-risk 17p deletion when treated outside the body. Preliminary results from the first study treating 9 relapsed/refractory CLL patients with 17p deletion with ABT-199 indicated that it may be beneficial in this unique patient population.
The purpose of this study is to evaluate how well ABT-199 works in patients with relapsed or refractory CLL who have 17p deletion. The safety and tolerability of ABT-199 will also be evaluated. In addition, substances found in the blood, bone marrow and tissue (biomarkers) that may indicate the effects or progress of the leukaemia and the activity of ABT-199 will be studied.
Participation in the study is expected to last approximately 10 months and includes about 25 visits to the research centre with post-treatment follow up every 12 weeks thereafter for 5 years. Blood tests, scans, bone marrow and lymph node biopsies and multiple patient questionnaires will be performed throughout the study. The study will include approximately 100 patients across Europe, North America and Australia. A pharmaceutical company is funding this study.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
13/EM/0340
Date of REC Opinion
30 Oct 2013
REC opinion
Further Information Favourable Opinion