Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)

Inclusion Criteria:

- Patients with high risk MDS or AML Exjade Pilot Assessing Hematologic Response in
AML/MDS UPCC# 40413

- Newly diagnosed acute myeloid leukemia, not a candidate for intensive induction
chemotherapy in the judgement of the investigator or unwilling to undergo intensive
induction chemotherapy OR

- AML that is refractory or relapsed after treatment with a non-intensive regimen, and
not a candidate for allogeneic transplant at this time OR

- MDS, IPSS 1.5 or greater, intolerant or with disease progression/lack of response to
hypomethylating agents.

- Age ≥ 18 years and able to provide independent informed consent

- Serum Ferritin: For enrollment on the study: serum ferritin ≥ 500 ng/mL at screening.
Samples must be obtained in the absence of concomitant infection. If transfusion is
scheduled, draw serum ferritin PRIOR to transfusion.

- ECOG performance status 0-2.

- Sexually active women must use an effective method of contraception, or must have
undergone clinically documented total hysterectomy and/or oophorectomy, or tubal
ligation or be postmenopausal (defined as amenorrhea for at least 12 months)

Exclusion Criteria:

- Prior therapy with intensive chemotherapy for AML

- Prior therapy with iron chelating agents within the last 6 months.

- Serum creatinine levels greater than 1.5x above the upper limit of normal

- AST or ALT levels greater than 5x the upper limit of normal

- Current therapy for AML or MDS (a ≥ 4 week washout period for any agent used to treat
AML or MDS prior to first dose of study drug is required).

- Current therapy with hydrea to control leukocytosis.

- Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the
absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal
range) infection. In the absence of clinical suspicion for active infection,
laboratory testing to assess Hepatitis B or C status will not be required at
screening.

- Clinical history of HIV positive test result (ELISA or Western blot). Laboratory
testing to assess HIV status will not be required at screening.

- Clinical or self reported history of drug or alcohol abuse within the 12 months prior
to enrollment

- ECOG Performance Status > 2

- Uncontrolled systemic hypertension

- Severe cardiac insufficiency (NYHA III or IV), with uncontrolled and/or unstable
cardiac or coronary artery disease not controlled by standard medical therapy

- Clinical diagnosis of or history of clinically relevant ocular toxicity related to
iron chelation

- Systemic diseases (cardiovascular, renal, hepatic, etc.) which in the opinion of the
investigator would prevent study treatment

- Pregnant or breast feeding women

- Treatment with systemic investigational drug within the past 4 weeks or topical
investigational drug within the past 7 days or plan to receive other investigational
drugs while participating in the study

- Other surgical or medical condition which might significantly alter the absorption,
distribution, metabolism or excretion of study drug

- History of non-compliance to medical regimens or patients who are considered
potentially unreliable and/or not cooperative

- Hematopoietic stem cell transplant for MDS or AML.

- Active CNS leukemia Patients who are found to be ineligible after screening procedures
will have the reason for ineligibility documented on the screening log. No further
data will be collected in the CRF for these patients.