Testing SP-420 Capsules to Remove Iron in Adults with Transfusion-Dependent Beta-Thalassemia or Low-Risk Myelodysplastic Syndromes Open-Label, Dose-Escalation Study for Up to 48 Weeks

What this Trial Is About

Researchers are studying SP-420, an oral medication, to understand its ability to remove excess iron from organs in people who have transfusion-dependent beta-thalassemia or low-risk myelodysplastic syndrome. The trial aims to find out how well SP-420 clears iron from the liver and how safe and tolerable different doses are for participants. This Phase 2 study is sponsored by Pharmacosmos AS and involves adults aged 18 and older who require regular transfusions and iron chelation therapy. Participants will take SP-420 capsules three times a week for 48 weeks across several study groups. The study includes dose escalation and dose-finding phases to evaluate different dosing levels of SP-420. Throughout the treatment period, participants will attend up to 20 clinic visits and undergo MRI scans to measure iron levels in the liver and other assessments to monitor safety and treatment effects. During the trial, researchers will track the dose-response relationship of SP-420 over the first 24 weeks for beta-thalassemia subjects and assess safety after 12 weeks in those with low-risk myelodysplastic syndrome. Additional monitoring includes measuring serum ferritin and ongoing safety checks over 48 weeks. Participants will be closely monitored for treatment tolerability and iron reduction, with regular visits and scans to evaluate outcomes and overall health.

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes