Phase 2 study in adults with HFE-related Hereditary Hemochromatosis
Research type
Research Study
Full title
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)
IRAS ID
1013195
Contact name
Joanna Rutecka
Contact email
Sponsor organisation
CSL Behring LLC
Research summary
Hereditary hemochromatosis (HH) is an inherited condition where iron levels in the body can get too high. The iron may build up in different places, like the pancreas, liver, heart, and joints, which can lead to diabetes, liver damage, heart disease, and arthritis.
Homeostatic iron regulator gene-related hereditary hemochromatosis (HFE-HH) is the most common type of HH. Current treatments, which involve drawing blood, may be unsuitable or not possible for some people and may cause disruption in people’s lives. The purpose of this study is to learn about the effects and safety of vamifeport in adult participants with HFE-HH.
Participants will take either vamifeport or placebo capsules by mouth twice daily for about one year. Participants will be randomly assigned to 1 of 4 groups, receiving either a low or high dose of vamifeport, or the matching placebo. Throughout the study, participants will undergo MRI scans, electrocardiograms (ECGs, to check heart activity), blood samplings, urine collections, physical examinations, and questionnaires about overall health and quality of life. Participants will temporarily stop standard HH procedures, such as regular blood collections, during the study. These procedures will be resumed, if medically required.
How much do iron levels in the liver change after a year of taking vamifeport? This will be assessed using magnetic resonance imaging (MRI), which is a medical imaging technique that uses magnets and radio waves to take detailed pictures of organs and tissues inside of the body, without using X-rays.
This study is for people who are at least 18 years of age, have a body mass index (a measure of weight relative to height, abbreviated as BMI) between 18.5 and 32 kg/m2, are diagnosed with HFE-HH and show signs of high levels of iron in their body
About 84 participants will take part in the study. Participants will be in the study for a total duration of about 1 year and 2 months.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
25/EE/0272
Date of REC Opinion
5 Feb 2026
REC opinion
Further Information Favourable Opinion