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

Status

Terminated

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02274233

Enrollment

Registered

2014-10-24

Start date

2014-10-31

Completion date

2015-09-30

Last updated

2015-09-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Beta-Thalassemia, Iron Overload

Keywords

Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation

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.

Interventions

DRUGSP-420

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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

Design outcomes

Primary

Measure	Time frame
Number of Participants with Adverse Events	Up to 35 days

Secondary

Measure	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

Countries

Canada, Lebanon, Thailand, Turkey (Türkiye), United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026