Phase 2 Randomized, Placebo-Controlled Study of the Efficacy and Safety of Vamifeport in Adults with HFE-Related Hereditary Hemochromatosis

The primary objective is to evaluate the efficacy of vamifeport on liver iron concentration (LIC) as measured by magnetic resonance imaging (MRI) in adults with hereditary hemochromatosis. 5 The secondary objectives include:

• Assessment of the safety and tolerability of the study intervention. 4

This clinical trial involves 31 adult participants diagnosed with hereditary hemochromatosis. The study population includes both male and female patients. Inclusion requires a confirmed diagnosis of the condition along with evidence of iron overload. Specific biochemical requirements include a transferrin saturation greater than 45% and serum ferritin levels between 200 ng/mL and 5000 ng/mL. Additionally, participants must exhibit a liver iron concentration between 3 and 16 mg/g dry weight as measured by magnetic resonance imaging. The body mass index of the subjects must be within the range of 18.5 to 32 kg/m².

This Phase 2, multicenter, randomized, placebo-controlled, double-blind study evaluates the efficacy and safety of vamifeport in adults with hereditary hemochromatosis. The primary objective is to assess the effect of the investigational product, CSL624, on magnetic resonance imaging (MRI)-based liver iron concentration (LIC). Participants undergo a screening visit to confirm eligibility through medical history, transferrin saturation (TSAT) levels, serum ferritin concentrations, and MRI-based LIC measurements. Following screening, subjects are assigned to receive either vamifeport or a matching placebo in a hard capsule form. The study design includes follow-up visits and an end-of-study visit to monitor clinical outcomes and safety parameters. The estimated recruitment period for the trial is from February 2026 to March 2028.

The experimental medication, CSL624, consists of the active substance vamifeport trihydrochloride. This agent is administered in a hard capsule pharmaceutical form via the oral route.

The control group receives a placebo, which is a capsule designed to match the appearance of the vamifeport formulation.

The efficacy of vamifeport in adult subjects with hereditary hemochromatosis will be evaluated. The primary endpoint is the change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC).