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

• Status: Terminated
• Trial end date: 2015-09-01
• Target enrollment: 0
• Participant gender: All

Summary

The 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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sideris Pharmaceuticals

Criteria

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