An Open-label, Dose-escalation, Dose-finding, and Proof-of-concept Trial of SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome. The main questions it aims to answer are: * How efficient is SP-420 in cleaning iron from the liver? * How is the safety and tolerability of ascending doses of SP-420? Participants will: * Take medication three times weekly * Attend up to 20 site visits * Undergo MRI scans
• Women and men aged 18 years or older
• Transfusion-dependent β-thalassemia including HbE/β-thalassemia requiring iron chelation therapy (β-thalassemia with mutation and/or multiplication of α-globin is allowed)
• On a stable dose of iron chelation for at least 4 weeks prior to screening
• Weight ≥ 35kg at screening
• Transfusion iron overload
• Treated and followed for at least the past 6 months in a specialized centre
• Women and men aged 18 years or older
• Very low, low, or intermediate risk Myelodysplastic Syndrome according to IPSS-R
• Weight ≥ 35kg at screening
• Transfusion iron overload
• Treated and followed for at least the past 6 months at medical facilities experienced with MDS