Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations
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|ClinicalTrials.gov Identifier: NCT04493138|
Recruitment Status : Recruiting
First Posted : July 30, 2020
Last Update Posted : August 25, 2020
|Condition or disease||Intervention/treatment||Phase|
|Chronic Myelomonocytic Leukemia Myelodysplastic Syndrome Myeloproliferative Neoplasm Recurrent Chronic Myelomonocytic Leukemia Recurrent Myelodysplastic Syndrome Recurrent Myeloproliferative Neoplasm||Drug: Azacitidine Drug: Quizartinib||Phase 1 Phase 2|
I. To determine the safety, tolerability and maximum tolerable dose (MTD) of quizartinib in combination with azacytidine.
II. To assess overall response (ORR) rate to quizartinib in combination with azacitidine.
I. To assess overall survival (OS), duration of response, leukemia-free survival (LFS), relapse-free survival (RFS) and safety profile.
II. Correlative studies.
OUTLINE: This is a phase I, dose-escalation study of quizartinib followed by a phase II study.
Patients receive azacitidine subcutaneously (SC) or intravenously (IV) over about 30 minutes on days 1-5 and quizartinib orally (PO) once daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||58 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/II Study of Azacitidine in Combination With Quizartinib for Patients With Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms With FLT3 or CBL Mutations|
|Actual Study Start Date :||July 21, 2020|
|Estimated Primary Completion Date :||February 3, 2022|
|Estimated Study Completion Date :||February 3, 2022|
Experimental: Treatment (azacitidine, quizartinib)
Patients receive azacitidine SC or IV over about 30 minutes on days 1-5 and quizartinib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Given SC or IV
- Overall response rate [ Time Frame: At 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 survival [ Time Frame: Time 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 response [ Time Frame: Duration 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 survival [ Time Frame: 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 ]Will be listed and summarized by the Kaplan-Meier estimator.
- Leukemia free survival [ Time Frame: Time 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) [ Time Frame: 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04493138
|Contact: Guillermo M. Bravoemail@example.com|
|United States, Texas|
|M D Anderson Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Guillermo M. Bravo 713-794-3604|
|Principal Investigator: Guillermo M. Bravo|
|Principal Investigator:||Guillermo M Bravo||M.D. Anderson Cancer Center|