ITCC-101/APAL2020D, A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML
What is the goal of the study?
Although the prognosis of children with acute myeloid leukemia (AML) has improved over the last decades, with overall survival (OS) rates approaching 70% as a result of intensive frontline treatment, aggressive salvage therapy following relapse and improvements in supportive care, outcome after relapse remains poor. The 4-year probability of survival (pOS) of children with AML in 1st relapse is 38% and the recommended treatment approach for first relapse includes an anthracycline-based re-induction followed by a second cycle of chemotherapy and HSCT, based on the results of the iBFM AML 2001/01 using FLAG-liposomal daunorubicin in 1st cycle, and FLAG in second cycle, followed by allogenic transplantation. In the iBFM AML 2001/01 study, one-year OS of early first relapse was 52% and late relapse of 66%. However, options for patients unable to tolerate anthracyclines in first relapse are limited. For patients beyond first relapse, limited data exist. The AML-BFM study group reported that survival of children with AML in 2nd relapse was poor, with a 5-year pOS of 15+/-4% and 31+/-9% following HSCT (n=25/73). Early second relapse (within one year after first relapse) was associated with dismal outcome (pOS 2+/-2%, n=44 vs. 33+/-9%, n=29; p<0.0001). there is no consensus on the therapy recommended in the 2nd relapses of aml in children. additional therapeutic options for children in 2nd relapsed aml, and children in 1st relapse unable to tolerate anthracycline, are needed. based on the promising results of the phase i ii venaml trial shown below, off-label use of venetoclax in children with relapsed aml in the united states is more and more frequent. a randomized trial of venetoclax in combination with intensive chemotherapy in children with 2nd relapsed aml and 1st relapsed aml unable to receive additional anthracycline would inform and evaluate if this agent is an effective option for this population to improve its poor prognosis.>0.0001).>
Who can participate in the study?
Please contact the study team listed below to learn more.