Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study



Status:Completed
Conditions:Blood Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:18 - Any
Updated:10/19/2018
Start Date:March 22, 2010
End Date:February 27, 2018

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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

The purpose of this study was to evaluate deferasirox and placebo with regard to event-free
survival (EFS) (a composite primary endpoint including death and non-fatal events related to
cardiac and liver function and transformation to AML) in patients with myelodysplastic
syndromes (low/int-1 risk) and transfusional iron overload.

A screening period lasting up to 35 days with two screening visits (Visit 1 and Visit 2 - at
least 14 days apart) was used to assess patient eligibility. Eligible patients were
randomized in a 2:1 ratio in deferasirox and placebo arms respectively. All patients who were
randomized in this study started study treatment at 10 mg/kg/day and could be titrated up to
40 mg/kg/day based on dose modification guidelines.

Patients who met a non-fatal event, as specified in the protocol, discontinued from the study
treatment and were followed for safety (28 days) and then evaluated with visits every three
months if they agreed to move into the post treatment evaluation phase. Subsequent to the
evaluation period, or at the end of treatment if a patient and treating physician decided
that a patient would not participate in the evaluation period, patients were followed by
phone monitoring every six months for their need for iron chelation therapy (ICT) and
survival follow-up (OS) until study end.

Patients who did not meet a non-fatal event continued study treatment as long as the patient
and the treating physician felt it was in the best interest for the patient or until the
study terminated. After termination of study treatment, all patients continued to be followed
for safety and endpoints during the evaluation period at visits occurring every three months.
Subsequent to the evaluation period, or at the end of treatment if a patient and treating
physician decided that a patient would not participate in the evaluation period, patients
were followed every 6 months for ICTs and OS.

Inclusion Criteria:

- Weigh between 35-135 kilograms

- Low or int-1 risk MDS

- Ferritin >1000 micrograms/liter at screening

- History of transfusion of 15 to 75 Packed Red Blood Cells (PRBC) units

- Anticipated to be transfused with at least 8 units of PRBCs annually during the study

- Women of child-bearing potential using effective methods of contraception during
dosing of study treatment

Exclusion Criteria:

- More than 6 months of cumulative ICT (such as daily deferasirox (Exjade®) or
deferiprone or 5×/week deferoxamine)

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

- Significant proteinuria

- History of hospitalization for congestive heart failure; other heart conditions as
specified in the protocol

- Systemic diseases which would prevent study treatment

- Hepatitis B; Hepatitis C; HIV

- Liver cirrhosis

- Pregnant, or breast-feeding patients, or patients of child-bearing potential not
employing an effective method of birth control

- History of drug or alcohol abuse within the 12 months prior to enrollment
We found this trial at
17
sites
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Anaheim, CA
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Chicago, IL
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Denton, Texas 76210
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Denton, TX
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Detroit, Michigan 48202
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Detroit, MI
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Greenwood Village, Colorado
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Greenwood Village, CO
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Hackensack, NJ
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Herston, Queensland
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Herston,
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Houston, TX
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Kalispell, MT
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Kansas City, Missouri 64131
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Kansas City, MO
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Las Vegas, NV
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Los Angeles, CA
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Manchester, Missouri 63021
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Manchester, MO
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from
San Antonio, TX
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Seattle, Washington 98107
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from
Seattle, WA
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Shreveport, Louisiana 71103
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from
Shreveport, LA
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from
Yakima, WA
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