Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

Inclusion criteria

• Transfusional iron overload due to: hereditary anemias such as sickle cell disease, β-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. Patients must also be transfusion-dependent and require chronic treatment with deferoxamine, deferasirox, and/or deferiprone.
• Willing to discontinue all existing iron chelation therapies throughout study period.
• Serum ferritin greater than 500 ng/mL at Screening.
• Baseline liver iron concentration and cardiac MRI T2* per protocol requirements.
• Mean of the previous three pre-transfusion hemoglobin concentrations greater than or equal to 7.5 g/dL.
• Agrees to use an approved method of contraception throughout study period.

Exclusion criteria

• As a result of medical review, physical examination or Screening investigations, the Principal Investigator considers the patient unfit for the study.
• Non-elective hospitalization within the 30 days prior to Baseline testing. (Patients with sickle cell anemia who are admitted to the hospital for management of sickle crisis pain whose uncomplicated hospital course was four days or less and who, 14 days prior to Baseline testing, have returned to their previous health status are acceptable.)
• Evidence of clinically relevant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow or skin disorder as determined by the Investigator.
• Evidence of significant renal insufficiency; possible examples include: serum creatinine above the upper limit of normal, proteinuria greater than 2 gm per day or calculated creatinine clearance of less than 60 mL/minute.
• Cardiac left ventricular ejection fraction outside of protocol requirements.
• Known sensitivity to magnesium stearate, croscarmellose sodium or FBS0701.
• Platelet count below 100,000/µL and/or absolute neutrophil count less than 1500/mm3 at Screening and <50% at Baseline testing by MRI
• Alkaline phosphatase, AST or ALT outside of protocol requirements.
• Liver Function Tests: ALT >5 times the local upper limit of normal on two occasions in the previous 12 months or ALT at Screening >200 IU/L
• Use of any investigational agent within the 30 days prior to the Baseline testing.