Dose Escalation of OXi4503 as Single Agent and Combination With Cytarabine w/Subsequent Ph 2 Cohorts for AML and MDS

Phase 1 will investigate maximum tolerated dose of OXi4503 as a single agent and in
combination with intermediate-dose cytarabine in subjects with relapsed/refractory AML or
MDS.

Phase 2 will investigate overall response rate of OXi4503 in combination with
intermediate-dose cytarabine in 1) subjects with MDS after failure of 1 prior
hypomethylating agent (Arm A) and 2) subjects with relapsed and refractory AML after
treatment failure of up to 1 prior chemotherapy regimen (Arm B).

Eligibility Criteria

Inclusion Criteria:

1. Provide informed consent

2. ≥ 18 years of age

3. Phase 1 (dose escalation) subjects must have either:

- AML that has failed to achieve complete remission or morphologic complete
remission or

- MDS - Marrow blasts must be > 5% and disease failed at least 1 prior
hypomethylating agent

4. Phase 2 (expansion) subjects must have either MDS or relapsed/refractory AML

5. Eastern Cooperative Oncology Group performance status 0, 1, or 2

6. Total bilirubin ≤ 2

7. Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels ≤
2.5 times upper limit of normal (ULN)

8. Serum creatinine < 2.5 times ULN

9. Prothrombin time (PT)/international normalized ratio and (PTT) in normal range ± 25%

10. Women of child-bearing potential

11. Males with female partners of child-bearing potential must agree to use
physician-approved contraceptive methods

Exclusion Criteria:

1. Acute promyelocytic leukemia

2. Absolute peripheral blood myeloblast count greater than 20,000/mm3

3. Uncontrolled hypertension

4. History of congenital long QT syndrome or torsades de pointes

5. Pathologic bradycardia or heart block

6. Prolonged baseline QTc

7. Hiistory of ventricular arrhythmia

8. Myocardial infarction and/or new ST elevation

9. Any history of hemorrhagic stroke

10. Symptomatic congestive heart failure

11. Major hemorrhagic event within 28 days

12. Suggestive central nervous system involvement with leukemia

13. Any open wound

14. Pregnant and nursing subjects are excluded

15. Treatment with any anticancer therapy

16. Treatment with colchicine is excluded.

17. Psychiatric disorders that would interfere with consent

Principal Investigator

Tara Lin, MD

Study Contact

Joyce Tungol 913-945-7547 jtungol2@kumc.edu

Estimated Completion Date

Sunday, October 1, 2017

ClinicalTrials.gov #

NCT02576301
09/13/2016