Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study of the Efficacy and Safety of Vamifeport in Adult Subjects With HFE-related Hereditary Hemochromatosis (FERROCLEAR Study)
CSL Behring
84 participants
Jan 22, 2026
INTERVENTIONAL
Conditions
Summary
This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary hemochromatosis (HFE-HH). The primary objective of the study is to assess the effect of vamifeport treatment on magnetic resonance imaging (MRI)-based liver iron concentration (LIC) in adult participants with HFE-HH.
Eligibility
Inclusion Criteria7
- Adult (≥ 18 years) and has provided written informed consent.
- Confirmed diagnosis of HFE-HH in medical history.
- Evidence of iron overload as shown by:
- TSAT \> 45% (confirmed at 2 visits, at least 14 days apart) at Screening; and
- Serum ferritin ≥ 200 nanogram per milliliter (ng/mL) and \< 5000 ng/mL (confirmed at 2 visits, at least 14 days apart) at Screening; and
- MRI-based LIC between 3 and 16 mg/g (53.7 and 286.5 millimol per kilogram \[mmol/kg\]) dry weight (dw) at Screening.
- Body mass index between 18.5 and 32 kilograms per meter squared (kg/m\^2).
Exclusion Criteria1
- Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.
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Interventions
Vamifeport capsule administered orally.
Placebo capsule matching IP administered orally.
Locations(96)
View Full Details on ClinicalTrials.gov
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NCT07332091