Safety of SP-420 in the Treatment of Transfusional Iron Overload

Inclusion Criteria

• •1. Age ≥18
• •2. Diagnosis of MDS or MF with transfusional iron overload
• •3. Patients with MDS, will include only those with MDS Revised international prognostic scoring system (IPSS-R) risk group of intermediate, high, or very high.
• •4. Patients with MF, will include only those with Dynamic International Prognostic Scoring System-Plus (DIPSS=Plus) risk category of intermediate-1, intermediate-2, and high risk.
• •5. Patients with sickle cell disease and transfusional iron overload
• •6. Not appropriate for other iron chelation therapy, per physician
• •7. Received 10 or more units of packed red blood cells in the preceding 24 months and remains red cell transfusion dependent
• •8. ECOG ≤ 3
• •9. ALT ≤ 3 times the upper limit of the normal range
• •10. Estimate glomerular filtration rate calculated using Cockroft Gault of ≥ 60 mL/min/1.73m2
• •11. Serum ferritin ≥1000 ng/ml
• •12. Willing to comply with all study procedures and be available for the duration of the study
• •13. Able to take oral medication and be willing to adhere to study medication for 28 days
• •14. Female patient must be post-menopausal (no menses for \> 12 consecutive months) or surgically sterile (i.e., bilateral oophorectomy, hysterectomy, or tubal sterilization; must agree to completely abstain for heterosexual intercourse; or, if sexually active, must agree to use 1 of the following methods for birth control from the date she signs the consent form until 30 days after final dose of the study drug.
• •* Progesterone implant
• •* Intrauterine device
• •* Combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method, partner with vasectomy conducted \>60 days before screening visit plus a hormone or barrier method
• •15. Male patients must agree to use 1 of the following methods for birth control from the date he signs the consent form until 30 days after final dose of the study drug: be surgically sterile by vasectomy conducted \> 60 days before screening visit plus use a barrier method, or, must agree to completely abstain from heterosexual intercourse, or must agree to use a combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method), or have a post-menopausal partner plus barrier method.