M15-531: ABT-199 in MDS
Research type
Research Study
Full title
A Phase 1b Dose Escalation Study Evaluating the Safety and Pharmacokinetics of Venetoclax in Combination with Azacitidine in Subjects with Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS).
IRAS ID
215945
Contact name
Ghulam Mufti
Contact email
Sponsor organisation
AbbVie Ltd
Eudract number
2016-001657-41
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 9 months, 20 days
Research summary
Myelodysplastic Syndrome (MDS) is a blood disorder that causes a drop in the number of healthy blood cells in a patient. MDS can be classified as either low, intermediate or high risk depending on the percentage of bone marrow that is affected, and whether or not just the red blood cells, or the red and white blood cells as well as the platelets are affected. Approximately half of patients with MDS have high risk MDS, which can develop into acute myeloid leukaemia, which has an average survival of one year with best supportive care.
One of the standard therapies for high risk MDS is the use of drugs called hypomethylating agents (HMAs), such as azacitidine. 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 is designed to determine if venetoclax added to azacitidine works better than azacitidine alone.
In this study, 2/3 of patients will receive venetoclax daily with standard dosing of azacitidine and the remaining 1/3 will receive azacitidine alone. This is a Phase 2, open-label, 3-arm randomised study in higher risk MDS patients who have not previously received treatment. AbbVie is funding this study which will enrol approximately 90 patients (30 patients per arm) at 50 sites worldwide.
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 MDS will be checked by taking blood and bone marrow, measuring side effects and by completing health questionnaires. Blood and bone marrow tests will be completed to see why some people respond better than others. Additional blood tests will be completed for genetic factors and to see how long the drug remains in the body.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
16/SC/0671
Date of REC Opinion
20 Dec 2016
REC opinion
Favourable Opinion