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A study to evaluate the effectiveness and safety of vamifeport in adults with hereditary hemochromatosis

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study is being conducted to evaluate the effectiveness and safety of a medication called vamifeport in adults with HFE-related hereditary hemochromatosis. This condition is a type of hereditary hemochromatosis, which is a genetic disorder that causes the body to absorb too much iron from food. Over time, this extra iron can build up in organs like the liver, potentially causing damage.

Participants in this study will be randomly assigned to receive either the experimental drug CSL624, which contains vamifeport trihydrochloride in a hard capsule form, or a placebo. The study is double-blind, meaning neither the participants nor the researchers know which treatment is being administered. During the study, the amount of iron in the liver will be monitored using MRI, a type of medical imaging that uses magnets to create detailed pictures of the inside of the body, to measure the liver iron concentration.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Study participation and medication administration

    You will be assigned to receive either the test medication, known as vamifeport, or a placebo, which is a capsule that looks like the medicine but contains no active ingredients. this process is double-blind, meaning neither you nor the medical staff will know which one is being taken during the study.

  2. Step 2

    Treatment monitoring

    The study aims to measure the effect of the vamifeport capsule on liver iron concentration, which is the amount of iron stored in the liver. this is measured using magnetic resonance imaging (mri), a type of medical scan that uses magnets to create detailed pictures of the inside of the body.

Who can join the trial?

7 criteria

  • You must be an adult, which means you are 18 years of age or older, and you must provide written agreement to participate in the study.
  • You must have a confirmed medical history of HFE-HH, which is a type of inherited condition where the body builds up too much iron.
  • You must show signs of iron overload, which means your body has more iron than it needs.
  • Your TSAT, which is a measurement of how much iron is currently moving through your blood, must be greater than 45% and confirmed during two separate visits at least 14 days apart.
  • Your serum ferritin level, which is a protein that stores iron in your body, must be between 200 ng/mL and 5000 ng/mL and confirmed during two separate visits at least 14 days apart.
  • Your MRI-based LIC, which is a measurement taken using a specialized scan to see how much iron is stored in your liver, must be between 3 and 16 mg/g.
  • Your body mass index (BMI), which is a calculation used to check if your weight is healthy for your height, must be between 18.5 and 32 kg/m².

Who cannot join the trial?

3 criteria

  • People who have laboratory abnormalities, which means results from blood or urine tests that fall outside of the normal range and could be a concern for your health.
  • People who have issues found during a 12-lead electrocardiogram (ECG), which is a medical test that records the electrical activity of your heart to check how it is working.
  • People with a medical history of health problems that could make participating in this study unsafe or affect the results.
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Investigated drugs

CSL624 is an experimental medicine being tested to see if it can help lower the amount of iron stored in the liver for adults with a condition called hereditary hemochromatosis.

What is already known about the treatment

Vamifeport trihydrochloride - This medication is taken by mouth in the form of a hard capsule. It is currently being studied in clinical trials and is not yet widely available as a standard treatment. It is used to treat hereditary hemochromatosis, a condition where too much iron builds up in the body. This drug works by blocking a specific protein in the intestine that helps absorb iron from food, thereby reducing the total amount of iron entering the bloodstream. This substance belongs to a class of drugs known as iron absorption inhibitors.

Investigated diseases

Hereditary hemochromatosis - This is a genetic condition that causes the body to absorb too much iron from the food eaten. Instead of being excreted, the excess iron builds up in various organs such as the liver, heart, and pancreas. Over time, this accumulation leads to increased iron concentrations within the tissues. The condition typically progresses as the body continues to collect iron faster than it can be utilized or removed. This ongoing buildup can gradually affect the normal function of the affected organs.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-523793-16-00Protocol codeCSL624_2001Estimated enrolment81 patientsSponsorCSL Behring LLC

sourced from the EU Clinical Trials Register and site verification

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