Azacitidine and Quizartinib for MDS or MDS/MPN with FLT3 or CBL Mutations
This study is looking at how safe and effective a combination of two drugs, azacitidine and quizartinib, is for people with myelodysplastic syndrome (MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN), including chronic myelomonocytic leukemia (CMML), who have specific genetic changes (FLT3 or CBL mutations). Azacitidine is a chemotherapy drug that helps stop cancer cells from growing, and quizartinib may also stop cancer cell growth by blocking certain enzymes. The study aims to find the best dose of quizartinib when given with azacitidine. To join, you must be at least 18 years old and have a diagnosis of MDS or MDS/MPN. For those not previously treated with similar drugs, your disease needs to be at a certain risk level or have more than 5% bone marrow blasts. The study will measure how many patients respond to the treatment, how long they live, and how long the treatment response lasts. The current recruitment status is unclear.
- Study design
- This is a Phase I/II interventional study with an estimated enrollment of 30 participants. It will first determine the best dose of quizartinib with azacitidine, then assess its effectiveness.
- What's involved
- Participants will receive azacitidine either under the skin or through a vein on days 1-5, and quizartinib by mouth once daily on days 1-28. These cycles repeat every 28 days as long as the treatment is working and well-tolerated.
- Compensation
- Not stated in the trial record.
- Follow-up
- After completing treatment, participants will be followed up at 30 days. Overall survival and duration of response will be assessed for up to 2 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Guillermo M Bravo · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
What this trial measures
- Overall response rateAt least 4 cycles of therapy in the absence of progression (1 cycle = 28 days)
Will be defined as complete remission, partial remission, complete remission with incomplete count recovery, marrow compete remission or hematological improvement. Will be estimated for all patients along with the 95% credible interval.
- Overall survivalTime from treatment start till death or last follow-up, assessed up to 2 years
Will be listed and summarized by the Kaplan-Meier estimator.
- Duration of responseDuration from the first documented onset of partial response or complete response to the date of progressive disease/relapse, assessed up to 2 years
Will be listed and summarized by the Kaplan-Meier estimator.
- Relapse-free survivalTime 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
Will be listed and summarized by the Kaplan-Meier estimator.
- Leukemia free survivalTime from treatment start to the time of progression to leukemia or death, assessed up to 2 years
Will be listed and summarized by the Kaplan-Meier estimator.
- Incidence of adverse events (AEs)Up to 2 years
The severity of the toxicities will be graded according to the latest version of National Cancer Institute Common Terminology Criteria for Adverse Events. The number and percent of subjects with treatment-emergent adverse events will be summarized according to intensity and drug relationship, and categorized by System Organ Class and preferred term by dose level/Part. All reported AEs that occur after signing informed consent will be included in the analysis of all reported AEs. Exposure to study drug and reasons for discontinuation of study drug will be tabulated.