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

Sideris Pharmaceuticals

Status and phase: Terminated; Phase 1

Conditions: Beta-Thalassemia; Iron Overload

Treatments: Drug: SP-420

Study type: Interventional

Funder types: Industry

Identifiers

NCT02274233

SP-420-702

Details and patient eligibility

About

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.

Enrollment: 24 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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24 participants in 6 patient groups

1.5 mg/kg

Experimental group

Description:

1.5 mg/kg SP-420 once daily for 14 days

Treatment:

Drug: SP-420

3 mg/kg

Experimental group

Description:

3 mg/kg SP-420 once daily for 14 days

Treatment:

Drug: SP-420

6 mg/kg

Experimental group

Description:

6 mg/kg SP-420 once daily for 14 days

Treatment:

Drug: SP-420

12 mg/kg

Experimental group

Description:

12 mg/kg SP-420 once daily for 14 days

Treatment:

Drug: SP-420

24 mg/kg

Experimental group

Description:

24 mg/kg SP-420 once daily for 28 days

Treatment:

Drug: SP-420

9 mg/kg

Experimental group

Description:

9 mg/kg SP-420 twice daily for 28 days

Treatment:

Drug: SP-420