Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myel… (NCT04493138) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations
United States30 participantsStarted 2020-07-21
Plain-language summary
This phase I/II trial studies the side effects and best dose of quizartinib when given with azacitidine and to see how well they work in treating patients with myelodysplastic syndrome or myelodysplastic/myeloproliferative neoplasm with FLT3 or CBL mutations. Chemotherapy drugs, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Quizartinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine and quizartinib may help to control myelodysplastic syndrome or myelodysplastic/myeloproliferative neoplasm.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥ 18 years as MDS is a very rare disease in the pediatric setting.
. Diagnosis of MDS or MDS/MPN including CMML according to WHO.
. For hypomethylating agent naïve patients: int-2 or higher by IPSS or \>5% bone marrow blasts if MDS or dysplastic CMML (WBC \<13x109/L). Patients with proliferative (WBC \>/= 13x109/L) CMML or MDS/MPN, or those with dysplastic CMML with high-risk molecular features (mutations in ASXL1, TP53 or more than 3 mutations) are also eligible independently of IPSS risk score or bone marrow blast percentage.
. For patients with prior hypomethylating agent therapy: no response after 6 cycles of azacitidine, decitabine, guadecitabine or ASTX727 or relapse or progression after any number of cycles.
. Detectable FLT3-ITD mutation in bone marrow and/or peripheral blood, or presence of CBL exon 8 or 9 deletions or point mutations.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Overall response rate
Timeframe: At least 4 cycles of therapy in the absence of progression (1 cycle = 28 days)
2
Overall survival
Timeframe: Time from treatment start till death or last follow-up, assessed up to 2 years
3
Duration of response
Timeframe: Duration from the first documented onset of partial response or complete response to the date of progressive disease/relapse, assessed up to 2 years
4
Relapse-free survival
Timeframe: Time from start of response to the date of event defined as the first documented progressive disease/relapse or death, whichever comes first, assessed up to 2 years
5
Leukemia free survival
Timeframe: Time from treatment start to the time of progression to leukemia or death, assessed up to 2 years
. Adequate hepatic function with total bilirubin \<2x ULN (will allow less than 5xULN if Gilbert's at investigator's discretion), AST or ALT \</=3xULN.
. ECOG Performance Status 0-2.
Exclusion criteria
. Uncontrolled infection not adequately responding to appropriate antibiotics.
. Screening ECG with a QTcF \>450 msec. The QTcF interval will be calculated by Fridericia's correction factor (QTcF). The QTcF will be derived from the average QTcF in triplicate. Patients are excluded if they have QTcF \>450. Subjects with prolonged QTcF interval in the setting of RBBB (right bundle branch block) may participate upon review and approval by the principal investigator and following evaluation by cardiology consult.
. Patients with congenital long QT syndrome.
. History or presence of sustained ventricular tachycardia requiring medical intervention.
. Any history of clinically significant ventricular fibrillation or torsades de pointes.
. Known history of second- or third-degree heart block (may be eligible if the patient currently has a pacemaker).
. Sustained heart rate of \<50/minute on screening ECG. The heart rate will be derived from the average heart rate in triplicate.
. Right bundle branch block + left anterior hemiblock (bifascicular block).