In short
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.
Key points
- Clinical trials of VAMIFEPORT TRIHYDROCHLORIDE are studying adults with HFE-related hereditary hemochromatosis, an inherited condition that can lead to too much iron in the body. The available trial is a Phase 2 interventional study with 81 participants. Researchers are mainly measuring liver iron concentration using MRI to see whether the treatment changes iron levels. The trial also includes a placebo comparison, which helps show whether any changes are linked to the study treatment. The study status is authorised.
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