Chandler

Researchers are evaluating vamifeport in adults with homeostatic iron regulator gene-related hereditary hemochromatosis HFE-HH, a condition characterized by iron overload. This phase 2, multicenter, randomized, placebo-controlled, double-blind study aims to assess the effect of vamifeport on liver iron concentration using magnetic resonance imaging MRI. The study focuses on adults with confirmed HFE-HH and iron overload to explore the potential impact of the treatment. Participants are randomly assigned to receive either a low dose or a high dose of vamifeport, or a placebo, all administered orally twice daily up to 360 days. The study compares these three groups over this treatment period to evaluate the treatments effect on liver iron levels. The study includes careful monitoring and assessment of safety and efficacy throughout the treatment duration. During the trial, participants undergo regular assessments including MRI scans to measure liver iron concentration at baseline and day 360. Safety is monitored by tracking adverse events, laboratory tests, and electrocardiograms up to day 390. Additional evaluations include measurements of transferrin saturation, serum ferritin, joint pain, fatigue, and quality of life questionnaires. Blood samples are collected to measure vamifeport concentrations at specific time points. Participants are followed for a total of about 13 months, including treatment and safety monitoring periods.