Safety & efficacy of AC220 in Pediatric and Young Adults with AML
Research type
Research Study
Full title
A PHASE 1/2, MULTI-CENTER, DOSE-ESCALATING STUDY TO EVALUATE THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF QUIZARTINIB ADMINISTERED IN COMBINATION WITH RE-INDUCTION CHEMOTHERAPY, AND AS A SINGLE-AGENT CONTINUATION THERAPY, IN PEDIATRIC RELAPSED/REFRACTORY AML SUBJECTS AGED 1 MONTH TO < 18 YEARS (AND YOUNG ADULTS AGED UP TO 21 YEARS) WITH FLT3-ITD MUTATION
IRAS ID
256900
Contact name
Brenda Gibson
Contact email
Sponsor organisation
Daiichi Sankyo, Inc.
Eudract number
2016-002919-18
Clinicaltrials.gov Identifier
74,552, IND NUMBER
Duration of Study in the UK
6 years, 11 months, 6 days
Research summary
The patients who will be taking party in this study have been diagnosed with acute myeloid condition has either not improved, or the AML has come back. The Patients have leukaemia cells that have a change in one of the genes (the part of the cell that contains the information about what a cell does). This change (or mutation) is called FLT3-ITD.
The main purpose of this study is to gather information about an investigational drug, called quizartinib that may help to treat AML. Quizartinib was designed to work against cancer cells that have FLT3-ITD. It is currently being tested in other studies to see if it works in adults with this type of AML.Children and young adults from 1 month old up to 21 years old may be part of this study. We expect that about 52 patients with AML may take part in this research study, which is being conducted at approximately 35 study sites around the world (in the United States and Europe).
There are 2 main parts to this study:Phase 1
The purpose of the first part is to find the right dose of quizartinib in children and young adults. At the start of the study, about 9 patients from 1 year old to 21. Based on side effects and blood test results, the dose may need to be adjusted. Additional patients may be treated with higher or lower doses to find a dose that is high enough to possibly be effective but without many severe side effects.
Once a dose for patients aged 1 to 21 years old is determined, patients from age 1 month will be able to enter the study. Similar testing and dose changes will be done to find a good dose for this age group.Phase 2
The purpose of the second part of the study is to test how effective quizartinib is at treating AML with FLT3-ITD. The doses chosen in Phase 1 will be used for patients in Phase 2.REC name
London - Hampstead Research Ethics Committee
REC reference
20/LO/0005
Date of REC Opinion
12 Mar 2020
REC opinion
Further Information Favourable Opinion