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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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City of Hope National Medical Center Los Angeles, California  91010
Mayo Clinic Jacksonville, Florida  32224
Rocky Mountain Cancer Centers Thornton, Colorado  80260
St. Agnes Healthcare Baltimore, Maryland  21229
The Ohio State University Columbus, Ohio  43210
Straub Clinic and Hospital Honolulu, Hawaii  96813
UCLA Medical Center Los Angeles, California  90095-7059
Roswell Park Cancer Center Buffalo, New York  14263
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Cedars-Sinai Medical Center, UCLA School of Medicine Los Angeles, California  90048
Emory University School of Medicine/Winship Cancer Institute Atlanta, Georgia  30322
University of Chicago Hospital Chicago, Illinois  60637-1470
Novartis Investigative Site Chicago, Illinois  60612
University of Kentucky College of Medicine, Markey Cancer Center Lexington, Kentucky  40536-0093
Cabrini Center for Cancer Care/Christus St. Frances Cabrini Hospital Alexandria, Louisiana  71301
Rush Cancer Institute Univ. of Massachussets Medical Center Worchester, Massachusetts  01605
Novartis Investigative Site Southfield, Michigan  48075
The Center for Cancer Care & Research (TCCCR) St. Louis, Missouri  63110
Oncology Hematology West, PC Omaha, Nebraska  68124-2346
The Cancer Center at Hackensack University Hackensack, New Jersey  07601
NMOHC Albuquerque, New Mexico  87109
Rochester General Hospital/Lipson Cancer and Blood Center Rochester, New York  14621
Wake Forest UniversitComprehensive Cancer Center Winston Salem, North Carolina  27157-1082
Novartis Investigative Site Portland, Oregon  97239
Thomas Jefferson University; Jefferson Medical College, Kimmel Cancer Center Philadelphia, Pennsylvania  19107
Western Pennsylvania Hospital Cancer Institute Pittsburgh, Pennsylvania  15224
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Baylor/The Methodist Hospital Houston, Texas  77030
Arlington Fairfax Hematology Oncology PC Arlington, Virginia  22205
Novartis Investigative Site Spokane, Washington  99202