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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 Using Serum Ferritin Monitoring

Phase 2
18 Years
Not Enrolling
Myelodysplastic Syndrome, 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 Using Serum Ferritin Monitoring

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.

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

- 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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

12 month safety in patients with myelodysplastic syndrome (MDS)

Outcome Time Frame:

throughout the study

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

May 2005

Completion Date:

March 2010

Related Keywords:

  • Myelodysplastic Syndrome
  • Iron Overload
  • ICL670
  • Deferasirox
  • Iron chelation
  • Chelator
  • Desferal
  • Myelodysplastic Syndromes
  • Preleukemia
  • Iron Overload



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