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NCT07332091
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
Conditions: Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Sex: All
Ages: 18 Years – N/A
Healthy volunteers: No
Phase: PHASE2
Enrollment: 84
Sponsor: CSL Behring
Location: Banner MD Anderson Gilbert Arizona
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 Criteria
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 2.4 and 16 mg/g (43.0 and 286.5 mmol/kg) dry weight (dw) at Screening.
* Body mass index between 18.5 and 34.9 kilograms per meter squared (kg/m\^2).
Exclusion Criteria:
* Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.
Source: ClinicalTrials.gov (NCT07332091). StuddyBuddy aggregates publicly available trial information.