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

Novartis

Status and phase

Completed

Phase 2

Conditions

Iron Overload

Myelodysplastic Syndrome

Treatments

Drug: Deferasirox

Study type

Interventional

Funder types

Industry

Identifiers

NCT00110266

CICL670AUS03

Details and patient eligibility

About

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.

Full description

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.

Enrollment

176 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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