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VAMIFEPORT TRIHYDROCHLORIDE

Clinical trials are investigating VAMIFEPORT TRIHYDROCHLORIDE in adults with HFE-related hereditary hemochromatosis, a condition linked to too much iron in the body. These studies aim to evaluate whether the treatment can improve liver iron levels and assess safety in the target patient group.

Table of Contents

Clinical trial overview

The available study of VAMIFEPORT TRIHYDROCHLORIDE is titled “Efficacy and safety of vamifeport in adult subjects with HFE-related hereditary hemochromatosis.”[1] It is an interventional trial, which means researchers are giving a study treatment and comparing results instead of only observing people.[1]

The trial is authorised and is in Phase 2.[1] Phase 2 studies usually focus on whether a treatment may help and continue to check safety in a specific patient group.

Who the study is for

This trial is for adult subjects with HFE-related hereditary hemochromatosis.[1] This condition is also described in the source as homeostatic iron regulator gene-related hereditary hemochromatosis.[1]

Hereditary hemochromatosis is an inherited iron disorder, meaning it runs in families and can lead to too much iron building up in the body. The study population is limited to adults, so children are not part of the trial description provided.

How the trial is designed

The study compares CSL624, listed as the trial drug, with a placebo capsule that matches vamifeport.[1] A placebo is a look-alike treatment with no active medicine, used so researchers can better see whether the study drug makes a difference.

The planned enrollment is 81 participants.[1] Enrollment means the number of people expected to join the study.

Main outcome being measured

The primary outcome is the change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC).[1] Baseline means the first measurement taken before treatment starts, and change from baseline means how much that number goes up or down later in the study.

MRI is a scan that creates pictures inside the body, and in this trial it is used to measure how much iron is in the liver without surgery.[1] Liver iron concentration, or LIC, is the amount of iron stored in the liver.[1]

The brief study summary says the purpose is to assess the effect of VAMIFEPORT TRIHYDROCHLORIDE treatment on MRI-based liver iron concentration in adults with HFE-related hereditary hemochromatosis.[1]

Important terms explained

Interventional study means the researchers actively give a treatment and then measure the results.[1]

Phase 2 means the study is past the first safety-only stage and is now looking more closely at possible benefit while still watching safety.

Placebo means an inactive capsule used for comparison so the study can be more reliable.[1]

Authorised means the trial has been approved to proceed.[1]

Trial ID Phase Condition studied Status Enrollment
2025-523793-16-00 Phase 2 HFE-related hereditary hemochromatosis Authorised 81

FAQ

  • What is being studied in VAMIFEPORT TRIHYDROCHLORIDE trials? The trial is studying whether VAMIFEPORT TRIHYDROCHLORIDE can reduce iron buildup in the liver. It also looks at safety in adults with HFE-related hereditary hemochromatosis.
  • Who can take part in these trials? The study is for adult subjects with HFE-related hereditary hemochromatosis. This means people must be adults and have this specific inherited iron disorder.
  • What phase is the trial? The trial is in Phase 2. Phase 2 studies usually look more closely at whether a treatment works and continue to check safety.
  • What condition is the trial focused on? The trial focuses on homeostatic iron regulator gene-related hereditary hemochromatosis, also called HFE-related hereditary hemochromatosis. This is an inherited condition that can cause too much iron to build up in the body.
  • What outcome is the trial measuring? The main outcome is the change in liver iron concentration measured by MRI. MRI is a scan that can measure iron in the liver without surgery.

Ongoing Clinical Trials on VAMIFEPORT TRIHYDROCHLORIDE

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

    Not yet recruiting

    2 1
    Investigated drugs:
    Austria Belgium Czechia France Germany Ireland +5

Glossary

  • Hereditary hemochromatosis: An inherited condition in which the body absorbs and stores too much iron.
  • HFE-related: Linked to the HFE gene, which helps control how the body handles iron.
  • Adult subjects: People who are old enough to be included as adults in a clinical study.
  • Phase 2: A trial stage that looks more closely at whether a treatment may work and keeps checking safety.
  • Interventional study: A study where researchers give a treatment or placebo and compare results.
  • Placebo: A look-alike treatment with no active medicine, used for comparison.
  • MRI: Magnetic resonance imaging, a scan that creates pictures inside the body and can measure liver iron.
  • Liver iron concentration (LIC): The amount of iron stored in the liver.
  • Change from baseline: The difference between the first measurement and the later measurement during the study.
  • Authorised: The study has been allowed to start by the proper authority.