A Phase II Study of Azacitidine and Sirolimus for the Treatment of High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia Refractory to or Not Eligible for Intensive Chemotherapy
PRIMARY OBJECTIVE:
I. To characterize the rate of response to azacitidine and sirolimus in adults with
high-risk myelodysplastic syndrome (MDS), or relapsed or refractory acute myeloid leukemia
(AML) or those unable or unwilling to tolerate high dose chemotherapy.
SECONDARY OBJECTIVES:
I. To determine the pharmacodynamic effect of sirolimus on inhibition of mammalian target of
rapamycin (mTOR) signaling in adults with high-risk MDS, or relapsed or refractory AML or
those unable or unwilling to tolerate high dose chemotherapy.
II. To determine the safety and tolerability of sirolimus and azacitidine in adults with
high-risk MDS, or relapsed or refractory AML or those unable or unwilling to tolerate high
dose chemotherapy.
III. To determine the progression free survival and overall survival in adults with
high-risk MDS, or relapsed or refractory AML or those unable or unwilling to tolerate high
dose chemotherapy.
IV. To determine if the quality of life of patients is improved with the combination of
azacitidine and sirolimus when compared to historical controls of azacitidine alone.
OUTLINE:
Patients receive sirolimus orally (PO) on days 1-10 or 1-12 and azacitidine intravenously
(IV) on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of
disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months.
Interventional
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Rate of response
MDS: Patients meeting an erythroid response, a platelet response, or a neutrophil response will be considered responders. AML: Patients achieving a complete remission (CR), complete response in the absence of a total platelet recovery (CRp), or partial remission (PR) will be considered responders.
Up to 5 years
No
Margaret Kasner, MD
Principal Investigator
Thomas Jefferson University
United States: Institutional Review Board
12D.587
NCT01869114
Name | Location |
---|---|
Thomas Jefferson University | Philadelphia, Pennsylvania 19107-6541 |