Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia

Inclusion Criteria:

- Subject has iron-overload secondary to β-thalassemia requiring chronic red blood cell
transfusions and iron chelation therapy

- Subject weighs ≥35 kg

- Subject is willing to discontinue current iron chelation therapy at least 7 days
prior to the first dose of SP-420 and for the duration of the study

- Serum ferritin ≥700 ng/mL and iron saturation ≥70% within 3 weeks before Baseline
(Day 1)

- Cardiac T2* score >20 msec within 6 months before Baseline (Day 1)

- Willing to use contraception during the study

Exclusion Criteria:

- Pregnant or breast-feeding

- Serum creatinine greater than the upper limit of normal

- Platelet count <100 × 10^9/L

- Use of another investigational drug within the last 30 days

- Significant cardiac, renal, hepatic dysfunction or other clinically significant
conditions that, in the opinion of the Investigator, would exclude the subject