Phase III Study of AG-221 in Late Stage AML with IDH2 mutation
Research type
Research Study
Full title
A PHASE 3, MULTICENTER, OPEN-LABEL,RANDOMIZED STUDY COMPARING THE EFFICACY AND SAFETY OF AG-221 (CC-90007) VERSUS CONVENTIONAL CARE REGIMENS IN OLDER SUBJECTS WITH LATE STAGE ACUTE MYELOID LEUKEMIA HARBORING AN ISOCITRATE DEHYDROGENASE 2 MUTATION
IRAS ID
181302
Contact name
Paresh Vyas
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2015-000344-42
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 11 months, 28 days
Research summary
Acute myeloid leukaemia (AML) is a form of cancer that is common in older patients. Mutations in the isocitrate dehydrogenase enzyme 2 (IDH2) have been found in approximately 15% of patients with AML.
The outcome of first line chemotherapy treatment is poor and many patients fail to attain complete remission (CR, ie refractory) or will eventually relapse. There is no single standard of care for relapsed or refractory AML. Since the prognosis is very poor there is a great need for new therapies.
Inhibition of IDH2 mutations may represent a promising targeted therapy for AML. AG-221 is designed to only block the IDH2 mutations. Data from the ongoing first-in-human study has shown AG-221 to be generally well tolerated and demonstrated CR in patients with IDH2 mutation positive relapsed or refractory AML.
The study purpose is to test the safety and efficacy of AG-221 compared with conventional care regimens (CCR), which include best supportive care (BSC) only, azacitidine plus BSC, low-dose cytarabine plus BSC or intermediate-dose cytarabine plus BSC, in patients with late stage AML refractory to or relapsed after second or third line therapy and positive for the IDH2 mutation. Patients will be randomly assigned to receive open-label tablets of AG-221 or one of the CCR on continuous 28-day treatment cycles. The trial duration is expected to be 28 months which includes 24 months enrollment, approximately 4 months treatment and a follow-up period.
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 IDH2 and assess treatment response. Bone marrow, blood, cheek swab samples will be used for genetic tests.
This study is being sponsored by Celgene Corporation. Approximately 280 participants will take part in the study, with approximately 11 to be randomised in the UK.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
15/EE/0442
Date of REC Opinion
11 Feb 2016
REC opinion
Further Information Favourable Opinion