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

Study Overview
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Brief Summary
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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.

Official Title
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Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia

Conditions
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Iron Overload, Beta-Thalassemia

Intervention / Treatment
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* Drug: SP-420


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1.5 mg/kg<br>1.5 mg/kg SP-420 once daily for 14 days | Drug: SP-420<br> <br> |
| Experimental: 3 mg/kg<br>3 mg/kg SP-420 once daily for 14 days | Drug: SP-420<br> <br> |
| Experimental: 6 mg/kg<br>6 mg/kg SP-420 once daily for 14 days | Drug: SP-420<br> <br> |
| Experimental: 12 mg/kg<br>12 mg/kg SP-420 once daily for 14 days | Drug: SP-420<br> <br> |
| Experimental: 24 mg/kg<br>24 mg/kg SP-420 once daily for 28 days | Drug: SP-420<br> <br> |
| Experimental: 9 mg/kg<br>9 mg/kg SP-420 twice daily for 28 days | Drug: SP-420<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Adverse Events |  | Up to 35 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Peak Plasma Concentration (Cmax) of SP-420 |  | Day 7 | 
| Area under the plasma concentration versus time curve (AUC) of SP-420 |  | Day 7 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation