Title
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia
Phase
Phase 1Lead Sponsor
Sideris PharmaceuticalsStudy Type
InterventionalStatus
TerminatedIndication/Condition
Iron Overload Beta-ThalassemiaIntervention/Treatment
sp-420 ...Study Participants
24The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted 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
