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A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of Deferasirox in Patients With Myelodysplastic Syndromes (Low/Int-1 Risk) and Transfusional Iron Overload

Phase 3
18 Years
Open (Enrolling)
Myelodysplastic Syndromes

Thank you

Trial Information

A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of Deferasirox in Patients With Myelodysplastic Syndromes (Low/Int-1 Risk) and Transfusional Iron Overload

Inclusion Criteria:

- Males or females ≥ 18 years of age

- Patients must weigh between 35-135 kg MDS low -int-1 risk as determined by IPSS score
and confirmed by bone marrow examination within 6 months prior to study entry

- Ferritin> 1000 mcg/L at screening

- History of 15 to 75 PRBC transfusions

- Anticipated to be transfused at least 8 times annually during the study

Exclusion Criteria:

- More than 6 months of cumulative iron-chelation therapy (such as daily deferasirox
(Exjade) or deferiprone or 5x/week deferosamine). intermittent deferoxamine doses in
association with blood transfusions are not exclusionary regardless of duration of
such treatment.

-- More than 3 years since patient began receiving regular transfusions (2 units per
8 weeks or 4 units received in a 3 month period).

- Creatinine clearance < 40 ml/min

- Serum creatinine >1.5x ULN at screening

- Significant proteinuria: urinary protein/creatinine ratio >0.5 mg/mg in a non first
void urine sample

- ECOG performance status > 2

- Left ventricular ejection fraction < 55% by ECHO

- History of hospitalization for Congestive Heart Failure

- Systemic disease that would prevent study treatment (uncontrolled hypertension,
cardiovascular renal, hepatic or metabolic disease)

- Hepatitis B or C (HBsAg in the absences or HBsAB or HCV Ab positive with HCV RNA

- History of HIV positivity by (ELISA or Western blot)

- Treatment with systemic investigational drug within 4 weeks or topical
investigational drug within 7 days of study start

- ALT or AST > 3.5 x ULN at screening

- Total bilirubin > 1.5 x ULN at screening

- Diagnosis of liver cirrhosis

- Patient participating in another clinical trial or receiving an investigational drug

- History of another malignancy within the past five years, with the exception of basal
skin carcinoma or cervical carcinoma in situ or completely resected colonic polyps
carcinoma in situ

- History of non-compliance with medical regimen, or patients potentially unreliable
and/or not cooperative

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

- Pregnant or intending to become pregnant or breast-feeding patents

- History of drug or alcohol abuse within the 12 months prior to enrollment.

- Other protocol-defined inclusion/exclusion criteria may apply

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Outcome Measure:

To compare deferasirox to placebo with regard to event-free survival (a composite primary endpoint including death and non-fatal events related to cardiac and liver function) in low and int-1 risk MDS patient with transfusional iron overload.

Outcome Time Frame:

1 year - 5 years

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

March 2010

Completion Date:

January 2018

Related Keywords:

  • Myelodysplastic Syndromes
  • MDS Study
  • Myelodysplastic Syndromes
  • Myelodysplastic Syndromes (low-int-1 risk)
  • Myelodysplastic Syndromes
  • Preleukemia



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