Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients

• Status: Completed
• Trial end date: 2008-03-28
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Chelating Agents; Deferasirox; Deferoxamine; Iron

Criteria

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

• Alanine aminotransferase (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)

• Eastern Cooperative Oncology Group (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 treatment

• 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