AG-120/AG-221 plus azacitidine in treatment of IDH1/IDH2 AML
Research type
Research Study
Full title
A PHASE 1B/2 OPEN-LABEL, RANDOMIZED STUDY OF 2 COMBINATIONS OF ISOCITRATE DEHYDROGENASE (IDH) MUTANT TARGETED THERAPIES PLUS AZACITIDINE: ORAL AG-120 PLUS SUBCUTANEOUS AZACITIDINE AND ORAL AG-221 PLUS SC AZACITIDINE IN SUBJECTS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA HARBORING AN IDH1 OR AN IDH2 MUTATION, RESPECTIVELY, WHO ARE NOT CANDIDATES TO RECEIVE INTENSIVE INDUCTION CHEMOTHERAPY
IRAS ID
201464
Contact name
Paresh Vyas
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2015-003951-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 10 months, 6 days
Research summary
Acute Myeloid Leukaemia (AML) is an aggressive cancer of the myeloid blood cell line with a poor prognosis. Induction Chemotherapy (IC) is the standard treatment for younger fit subjects but those subjects considered ineligible for IC due to old age or other complications have an especially poor prognosis with a mean survival of 2-8 months. Treatment with Azacitidine has increased the survival rate of this group to 9-10 months but there is a significant medical need for a new therapy to prolong survival of these subjects.
Two mutant proteins IDH1 and IDH2 have been identified in some cancers including AML. The study drugs, AG-120 and AG-221, inhibit these two mutant proteins. Preliminary clinical data from ongoing Phase 1 studies show these drugs are generally well tolerated and demonstrate an improvement in survival and complications of this group of subjects.
This open-label Phase 1b/2 randomised trial sponsored by Celgene Corportation is designed to investigate the safety, tolerability and efficacy of AG-120 or AG-221 combined with azacitidine versus azacitidine alone in subjects with AML that harbour the IDH1 or IDH2 mutations respectively, who are not candidates to receive intensive induction chemotherapy.
Approximately 165 worldwide will be enrolled into this study. The UK is taking part in the Phase 2 part of the study only. Subjects are expected to be in the Phase 2 part of the study for up to 30 months.
Study procedures include: vital signs, physical exams, ECGs, ECHO/MUGAs, urine/blood samples, bone marrow aspirates and/or biopsies and peripheral blood to test for IDH1 and IDH2 and assess treatment response. Bone marrow, blood, and cheek swab samples will be used for genetic tests.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
16/SC/0231
Date of REC Opinion
8 Aug 2016
REC opinion
Further Information Favourable Opinion