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
Study on the effects of investigational medication for hereditary hemochromatosis
Eric Orman
Primary Investigator
Brief description of study
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.
Detailed description of study
- Drug: Vamifeport
- Drug: Placebo
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: HFE-related Hereditary Hemochromatosis, HFE-HHHomeostatic Iron Regulator Gene-related Hereditary Hemochromatosis,
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Age: 18 years - 100 years
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Gender: All
Inclusion Criteria:
- 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 Criteria:
- Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.
This study investigates the effects of an investigational medication on hereditary hemochromatosis, a condition where the body absorbs too much iron from food, leading to excess iron storage in organs. The purpose of this study is to evaluate how the investigational medication affects liver iron concentration (LIC) in adults with this condition.
Participants will receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine. The study will use MRI scans to monitor changes in liver iron concentration and assess the safety and tolerability of the treatment.
- Who can participate: Adults aged 18 and older with a diagnosis of hereditary hemochromatosis are eligible to participate. Key eligibility factors include stable liver function and no history of liver transplantation.
- Study details: Participants will either take the investigational medication or a placebo. MRI scans will be used to check liver iron levels during the study.
