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

Phase 1

Terminated

• Conditions: Iron Overload; Beta-Thalassemia
• Interventions: Drug: SP-420

• Registration Number: NCT02274233
• Lead Sponsor: Sideris Pharmaceuticals

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-10
• Study First Submitted: 2014-10-21
• Study First Submitted QC: 2014-10-22
• Study First Posted: 2014-10-24
• Status Verified: 2015-09
• Primary Completion: 2015-09
• Study Completion: 2015-09
• Last Update Submitted: 2015-09-28
• Last Update Posted: 2015-09-29

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 24

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
9 mg/kg	SP-420	9 mg/kg SP-420 twice daily for 28 days
6 mg/kg	SP-420	6 mg/kg SP-420 once daily for 14 days
3 mg/kg	SP-420	3 mg/kg SP-420 once daily for 14 days
1.5 mg/kg	SP-420	1.5 mg/kg SP-420 once daily for 14 days
24 mg/kg	SP-420	24 mg/kg SP-420 once daily for 28 days
12 mg/kg	SP-420	12 mg/kg SP-420 once daily for 14 days

Primary Outcome Measures

Name	Time	Method
Number of Participants with Adverse Events	Up to 35 days

Secondary Outcome Measures

Name	Time	Method
Area under the plasma concentration versus time curve (AUC) of SP-420	Day 7
Peak Plasma Concentration (Cmax) of SP-420	Day 7

Trial Locations

Locations (1)

Sideris Investigative Site; 🇹🇷 Izmir, Turkey