Actively Recruiting
Phase II Study of Quizartinib Combined with Chemotherapy and as Single-agent After High Dose Therapy in Newly Diagnosed Pediatric FLT3-ITD+ and NPM1 Wild-type AML Patients
Led by Princess Maxima Center for Pediatric Oncology · Updated on 2025-08-05
60
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
Sponsors
P
Princess Maxima Center for Pediatric Oncology
Lead Sponsor
D
Daiichi Sankyo
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating a treatment approach for children newly diagnosed with acute myeloid leukemia (AML) who have specific genetic markers: a FLT3-ITD mutation and NPM1 wild-type. This phase II, open-label study aims to improve cure rates while reducing unnecessary side effects. The study is linked to a master protocol and focuses on the safety, effectiveness, and how the body processes quizartinib, a drug targeting the FLT3 mutation. Participants will receive quizartinib combined with standard chemotherapy during induction and consolidation phases. Quizartinib is given orally once daily for 14 days following chemotherapy cycles, which range from 3 to 5 cycles. After high dose chemotherapy or stem cell transplantation, patients continue quizartinib treatment for six 28-day courses to evaluate its effects as a single agent. Throughout the study, children will undergo various evaluations including safety monitoring and effectiveness assessments during the induction courses. The research team will track adverse events, laboratory results, and drug levels up to 1.5 years, with some efficacy outcomes followed for up to 3 years. Participants' health will be regularly assessed through clinical exams, laboratory tests, and performance status scores to monitor their response and safety during the trial period.
CONDITIONS
Brief Title
CHIP-AML22/Quizartinib: Quizartinib + Chemotherapy in Newly Diagnosed Pediatric FLT3-ITD+ and NPM1wt AML Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Enrolled in the CHIP-AML22/Master protocol before joining this linked trial
- Confirmed presence of FLT3-ITD mutation and NPM1 wild-type by local lab testing
- Age between 1 month and 18 years at initial diagnosis
- Karnofsky performance status over 50% if older than 16 years
- Lansky performance status over 50% if 16 years or younger
- Adequate kidney function with eGFR ≥ 50 mL/min/1.73 m2
- Adequate liver function with bilirubin less than 1.5 times upper limit (up to 5 times if related to leukemia) and AST/ALT less than 5 times upper limit (up to 10 times if related to leukemia)
- Life expectancy greater than 6 weeks
- Negative pregnancy test for girls at or beyond menarche within 2 weeks before enrollment
- Able to swallow quizartinib or receive it by feeding tube
- Written informed consent or assent obtained according to local laws
You will not qualify if you...
- Patients with only extramedullary leukemia disease
- Uncontrolled or significant heart diseases including congenital long QT syndrome, serious arrhythmias, QTc interval over 450 ms, low heart pumping function, recent heart attack or angina, severe heart block, very low heart rate, uncontrolled high blood pressure, left bundle branch block, or advanced heart failure
- Known HIV infection or active serious liver disease such as hepatitis B or C
- Gastrointestinal diseases that affect drug absorption
- Use of strong or moderate CYP3A inducers during the study
- History of allergic reaction to study drugs or their ingredients
- Serious illnesses that prevent completing study treatment
- Participation in other studies that interfere with this trial's goals
- For safety run-in phase: patients with severe central nervous system leukemia (CNS3) or using strong CYP3A4 inhibitors unless they can stop before enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 6 months covering induction, consolidation, and continuation courses
Participants receive quizartinib combined with standard chemotherapy across induction and consolidation courses. Quizartinib is taken orally once daily for 14 consecutive days during each induction and consolidation course, followed by continuation treatment with quizartinib for six 28-day courses after high dose chemotherapy or stem cell transplant if applicable.
Multiple visits during induction and consolidation courses, followed by regular visits during continuation treatment
Duration - Up to 1.5 years
Participants are monitored for safety, efficacy, and pharmacokinetics after treatment completion for up to 1.5 years.
Visits at multiple time points during follow-up
Trial Site Locations
Total: 1 location
1
Princess Máxima Center for pediatric oncology
Utrecht, Utrecht, Netherlands, 3584 CS
Actively Recruiting
Research Team
R
Renske Benedictus
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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