Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis

Plain-language summary

This study evaluates the efficacy and safety of the drug Vamifeport compared with placebo in adults with HFE gene-related hereditary hemochromatosis. It can enroll people aged 18 and over with a confirmed diagnosis of HFE-HH and evidence of iron overload. The study is being carried out in Phase 2 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Recruiting

Sponsor

CSL Behring

Intervention

Vamifeport, Placebo

Start date

1/22/2026

Estimated completion

4/6/2028

Countries

United States, Australia, Austria, Belgium, Canada, Czechia, Denmark, France, Germany, Ireland, Italy, Netherlands, New Zealand, Poland, Romania, Spain, Switzerland, United Kingdom

Related genes

Locations

• Gilbert, United States
• San Diego, United States
• San Diego, United States
• Jacksonville, United States
• Indianapolis, United States
• Baton Rouge, United States
• Baltimore, United States
• Bethesda, United States
• Frederick, United States
• Ann Arbor, United States
• Duluth, United States
• Flemington, United States
• Chapel Hill, United States
• Durham, United States
• Oklahoma City, United States
• York, United States
• Murray, United States
• Seattle, United States
• Spokane, United States
• Brisbane, Australia
• Chandler, Australia
• Clayton, Australia
• Perth, Australia
• Westmead, Australia
• Innsbruck, Austria
• Linz, Austria
• Vienna, Austria
• Edegem, Belgium
• Ghent, Belgium
• Jette, Belgium
• Liège, Belgium
• Yvoir, Belgium
• Calgary, Canada
• Hamilton, Canada
• Winnipeg, Canada
• Brno, Czechia
• Ostrava, Czechia
• Prague, Czechia
• Aarhus, Denmark
• Copenhagen, Denmark

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Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.