Venetoclax in children with relapsed AML
Research type
Research Study
Full title
A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML
IRAS ID
1005343
Contact name
Trial and Data Centre N/A
Contact email
Sponsor organisation
Princess Máxima Center
Eudract number
2021-003212-11
Clinicaltrials.gov Identifier
Research summary
We will investigate whether adding the medicine venetoclax to standard chemotherapy leads to improved survival for children and young adults with relapsed acute myeloid leukaemia (AML). The prognosis of children with AML has improved over the last decades, but the outcome after relapse remains poor. Most of the chemotherapy regimens include anthracyclines that can cause damage to the heart in some children. Therefore, we will investigate in this study whether adding venetoclax to anthracycline-free chemotherapy leads to improved survival for children and young adults with relapsed AML. Venetoclax is a small molecule that binds to, and triggers the death of, cancer cells.
The study is open to children and young adults who have had disease recurrence for the first time and may have compromised heart function, and therefore do not tolerate anthracyclines anymore, and children and young adults who have disease recurrence for the second time. All participants will be treated with 1 or 2 cycles of chemotherapy, consisting of medications called fludarabine and cytarabine (FLA). Participants whose AML cells have certain characteristics (called CD33-positive) will also receive the medicine gemtuzumab ozogamicin (GO).
For this study, venetoclax will be added to the treatment of half of the participants. This will be determined by a process called randomisation – meaning the assignment to the treatment with or without venetoclax is made by chance.
The treatment with the first 2 cycles will last about 10-12 weeks in total. The duration of the post-treatment period varies by participant. During the study, participants will undergo procedures such as; physical medical examination, questionnaires, scans (i.e. X-ray, CT, MRI), heart tests (electrocardiograms), blood sampling, collection of bone marrow, and lumbar punctures. After treatment has started, we would like to follow each participant for another 5 years.
REC name
London - City & East Research Ethics Committee
REC reference
23/LO/0767
Date of REC Opinion
29 Sep 2023
REC opinion
Further Information Favourable Opinion