Acute Myeloid Leukemia
Conditions
Brief summary
Assessment of safety and tolerability of drug combination and determine time on treatment, Overall survival (OS) and response rate with patient disease burden, and type of disease
Interventions
DRUGADI-PEG 20
Investigational Medicine
DRUGCytarabine
low-dose cytarabine 20 mg BID twice daily for 10 days, every 28 days
Sponsors
Polaris Group
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
17 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. AML diagnosed by morphologic (with \>20% blasts in blood or bone marrow) and histochemical and/or cell surface marker criteria. 2. Patients with AML must fall into one of the following: 1. Patients with AML (i.e., \> 20% bone marrow blasts) who are deemed unfit\* for intensive chemotherapy with refractory or relapsed disease. The patients must have been refractory to at least one cycle of cytarabine containing regimens or at least two cycles of azacitidine or similar hypomethylating agents. 2. Patients with untreated AML (i.e., \> 20% bone marrow blasts) with intermediate risk karyotype (MRC risk group) who are deemed unfit for intensive chemotherapy. 3. Patients with untreated AML with adverse risk karyotype (MRC risk group) who are deemed unfit for intensive chemotherapy and who are intolerant of azacitidine (or other hypomethylating agents) or who are unable to access azacitidine or other hypomethylating agents. * Patients unfit for conventional intensive chemotherapy are defined as having at least one of the following based on the conceptual criteria of Ferrara (2013): 1. Advanced age (over 75 years). 2. Cardiac impairment with ejection fraction ≤ 50% or heart failure (NYHA class 2) or ischemic heart disease with stable angina. 3. Pulmonary impairment: chronic obstructive pulmonary disease (COPD) stage 1-2 (forced expiratory volume in one second \[FEV1\] \> 49%) or other comparable respiratory disease with forced vital capacity (FVC) \> 50%. 4. Hepatic comorbidity with Child-Pugh grade A cirrhosis 5. Chronic kidney disease stage 3 but with creatinine clearance \> 30 mls/min 6. Any other comorbidity that the physician judges to be incompatible with intensive chemotherapy. 3. Age \> 17 years. 4. ECOG performance status of 0-2. 5. Bone marrow aspirate and/or biopsy for testing for ASS1-deficiency. This must be a fresh sample obtained after any prior chemotherapy and before enrollment in this study. ASS1-deficiency is not required for study entry, but the fresh bone marrow sample must be processed either before or within 1 week of first study dose (see Section 10 for more details).
Exclusion criteria
A subject will not be eligible for study participation if he/she meets any of the
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | through study completion; anticipated to be 2 years |
Countries
Taiwan
Outcome results
None listed