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An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-Risk and INT-1 Myelodysplastic Patients

Phase 2
18 Years
Not Enrolling
Myelodysplastic Syndromes, Iron Overload

Thank you

Trial Information

An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-Risk and INT-1 Myelodysplastic Patients

Patients will be screened for eligibility to determine if they meet all inclusion/exclusion
criteria. The screening period will be up to 4 weeks. Patient's baseline LIC will be
determined non-invasively by means of MRI R2 analysis. In addition, blood and urine samples
will be taken for the determination of baseline safety data.

Inclusion Criteria:

- Male or female patients with low or intermediate (INT-1) risk MDS, determined via
IPSS criteria, with transfusional iron overload. NOTE: Bone marrow morphology and
cytogenetic studies completed within 3 months prior to screening can be used if the
patient has been hematologically stable. Every attempt to obtain cytogenetics studies
should be made; however, if there is culture failure, repeat marrow aspiration will
not be mandated. In this case, RAEB with less than 11% marrow blasts will be

- Patients can be EITHER naïve to iron chelation OR have had prior treatment with
deferoxamine (DFO). DFO must be discontinued the day prior to starting deferasirox

- Age: greater than or equal to 18 years

- Serum ferritin:

- For entry into the screening period: serum ferritin greater than or equal to
1000 µg/mL on at least two occasions, at least two weeks apart, during the prior
year. Samples must be obtained in the absence of concomitant infection;

- For enrollment into the study: serum ferritin greater than or equal to 1000
µg/mL at screening (via the central lab) obtained in the absence of concomitant

- A lifetime minimum of 30 previous packed red cell transfusions

- Life expectancy greater than or equal to 6 months

- Women must have a negative serum or urine pregnancy test and use an effective method
of contraception, or must have undergone clinically documented total hysterectomy
and/or oophorectomy, or tubal ligation or be postmenopausal (defined by amenorrhea
for at least 12 months).

- Able to provide written informed consent

Exclusion Criteria:

- Serum creatinine above the upper limit of normal

- ALT greater than 250 U/L during screening

- 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

- Significant proteinuria as indicated by a urinary protein/creatinine ratio greater
than 0.5 mg/mg in a non-first void urine sample during screening (or alternatively in
two of three samples obtained for screening)

- History of HIV positive test result (ELISA or Western blot)

- ECOG performance status greater than 2

- Uncontrolled systemic hypertension

- Unstable cardiac disease not controlled by standard medical therapy

- Third degree atrioventricular (AV) block or QT interval prolongation above the normal

- History of clinically relevant ocular toxicity related to iron chelation

- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study

- Pregnancy or breast feeding

- Treatment with a systemic investigational drug within the past 4 weeks or a topical
investigational drug within the past 7 days.

- Other surgical or medical condition which might significantly alter the absorption,
distribution, metabolism or excretion of any drug. The investigator should be guided
by evidence of any of the following:

- inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal

- major gastrointestinal tract surgery, such as gastrectomy, gastroenterostomy, or
bowel resection;

- pancreatic injury or pancreatitis or indications of impaired pancreatic
function/injury, as indicated by abnormal lipase or amylase;

- urinary obstruction or difficulty in voiding.

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

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety and tolerability in myelodysplastic syndrome (MDS) patients

Outcome Time Frame:

12 months

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

April 2005

Completion Date:

January 2008

Related Keywords:

  • Myelodysplastic Syndromes
  • Iron Overload
  • MDS
  • Myelodysplastic Syndrome
  • ICL-670
  • ICL-670 and Myelodysplastic Syndrome
  • Myelodysplastic Syndromes
  • Preleukemia
  • Iron Overload



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Stanford University Medical Center Stanford, California  94305-5408
Karmanos Cancer Center Detroit, Michigan  48201