The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.

Condition	Treatment or Intervention	Phase
Myelodysplastic Syndrome Iron Overload	Drug: Deferasirox (Exjade, ICL670, ICL-670)	Phase II

Further Study Details: 

Primary Outcomes: Safety and tolerability
Secondary Outcomes: Efficacy of deferasirox based on changes in serum ferritin; To evaluate the role of NTBI (LPI and DCI), serum iron, transferrin and transferrin saturation; To evaluate change in transfusion requirements; To evaluate trough pharmacokinetics; To assess drug accountability; To explore the utility of magnetic resonance imaging (MRI)
Expected Total Enrollment:  150

Study entry requires a diagnosis of low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria and serum ferritin ≥ 1000 ng/mL. Patients must have had at least 30 prior red blood cell transfusions. Deferasirox will be administered at an initial dose of 20 mg/kg orally once per day. Patient transfusion history and at least three complete blood count (CBC) values must be available for the 12 weeks prior to study registration for patients with MDS and chronic iron overload from blood transfusions.

Ages Eligible for Study:  18 Years and above,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• Male or female patients with low or intermediate (INT-1) risk MDS
• Patients can be EITHER naïve to iron chelation OR have had prior treatment with deferoxamine (DFO).
• Age greater than or equal to 18 years
• Availability of transfusion records for the 12 weeks prior to registration
• A lifetime minimum of 30 previous packed red blood cell transfusions
• Availability of at least three CBC values (pretransfusion) during the 12 weeks prior to registration
• Serum Ferritin: For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year. Serum ferritin ≥ 1000 ng/mL at screening via the central lab.
• Life expectancy ≥ 6 months
• 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)
• Able to provide written informed consent

Exclusion Criteria:

• Serum creatinine above the upper limit of normal
• ALT > 500 U/L during screening
• Clinical or laboratory evidence of active Hepatitis B or C
• Urinary protein/creatinine ratio > 0.5 mg/mg
• History of HIV positive test result (ELISA or Western blot)
• ECOG Performance Status > 2
• Patients with uncontrolled systemic hypertension
• Unstable cardiac disease not controlled by standard medical therapy
• Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
• Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
• Pregnancy or breast feeding
• Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
• 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

Please refer to this study by ClinicalTrials.gov identifier  NCT00110266

Novartis Oncology Clinical Trials Call Center      1-800-340-6843 

Alabama
      Local Institution, Birmingham,  Alabama,  35294,  United States
Arizona
      Local Institution, Tucson,  Arizona,  85724-5024,  United States
      Local Institution, Scottsdale,  Arizona,  85259,  United States
California
      Local Institution, Los Angeles,  California,  90048,  United States
      Local Institution, San Francisco,  California,  94143-0324,  United States
      Local Institution, Duarte,  California,  91010,  United States
Colorado
      Local Institution, Aurora,  Colorado,  80012,  United States
Florida
      Local Institution, Tampa,  Florida,  33612,  United States
      Local Institution, Jacksonville,  Florida,  32224,  United States
Georgia
      Local Institution, Atlanta,  Georgia,  30322,  United States
Illinois
      Local Institution, Chicago,  Illinois,  60611-2927,  United States
      Local Institution, Chicago,  Illinois,  60637-1470,  United States
      Local Institution, Chicago,  Illinois,  60612,  United States
Indiana
      Local Institution, Indianapolis,  Indiana,  46260,  United States
Kentucky
      Local Institution, Lexington,  Kentucky,  40536-0093,  United States
Maryland
      Local Institution, Baltimore,  Maryland,  21231-1000,  United States
      Local Institution, Baltimore,  Maryland,  21229,  United States
Massachusetts
      Local Institution, Worchester,  Massachusetts,  01605,  United States
      Local Institution, Boston,  Massachusetts,  02115-6084,  United States
Minnesota
      Local Institution, Rochester,  Minnesota,  55905,  United States
Missouri
      Local Institution, St. Louis,  Missouri,  63110,  United States
      Local Institution, Kansas City,  Missouri,  66160,  United States
Nebraska
      Local Institution, Omaha,  Nebraska,  68198-7680,  United States
New Hampshire
      Local Institution, Lebanon,  New Hampshire,  03756-0001,  United States
New Jersey
      Local Institution, Hackensack,  New Jersey,  07601,  United States
New York
      Local Institution, Rochester,  New York,  14642,  United States
      Local Institution, Buffalo,  New York,  14623,  United States
      Local Institution, New York,  New York,  10021,  United States
      Local Institution, New York,  New York,  10021,  United States
      Local Institution, Rochester,  New York,  14621,  United States
North Carolina
      Local Institution, Winston Salem,  North Carolina,  27157-1082,  United States
      Local Institution, Durham,  North Carolina,  27710,  United States
Ohio
      Local Institution, Columbus,  Ohio,  43210,  United States
      Local Institution, Cleveland,  Ohio,  44195,  United States
Oregon
      Local Institution, Portland,  Oregon,  97201,  United States
Pennsylvania
      Local Institution, Philadelphia,  Pennsylvania,  19107,  United States
      Local Institution, Philadelphia,  Pennsylvania,  19104,  United States
      Local Institution, Pittsburgh,  Pennsylvania,  15224,  United States
Tennessee
      Local Institution, Nashville,  Tennessee,  37232-5505,  United States
Texas
      Local Institution, Houston,  Texas,  77030,  United States
Virginia
      Local Institution, Arlington,  Virginia,  22205,  United States

Study ID Numbers:  CICL670AUS03
Record last reviewed:  May 2005
Last Updated:  May 4, 2005
Record first received:  May 4, 2005
ClinicalTrials.gov Identifier:  NCT00110266
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-05-17