Vamifeport for Hemochromatosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called vamifeport for individuals with hereditary hemochromatosis, a condition where the body absorbs excessive iron. Researchers aim to determine if vamifeport can reduce liver iron levels, as measured by MRI scans. Participants will receive either a low dose, a high dose, or a placebo for up to 360 days. The trial seeks adults diagnosed with hereditary hemochromatosis who show signs of iron overload. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group.
Do I have to stop taking my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that vamifeport is likely to be safe for humans?
Research has shown that vamifeport is generally safe for people. In one study, participants took vamifeport pills in doses of either 60 mg or 120 mg, once or twice a day, and most did not experience serious side effects. The study found no major unexpected medical problems. These results suggest that vamifeport might be safe for individuals with hereditary hemochromatosis, a condition where the body absorbs too much iron. It is important to note that these findings come from earlier research, and ongoing studies continue to assess its safety and effectiveness.12345
Why do researchers think this study treatment might be promising for hemochromatosis?
Most treatments for hemochromatosis, like phlebotomy (removing blood) or chelation therapy (using drugs to remove excess iron), focus on reducing iron levels in the body. But Vamifeport works differently by targeting iron metabolism at a deeper level. Researchers are excited about Vamifeport because it’s an oral medication that could offer a more convenient and less invasive option compared to current treatments. By potentially regulating iron at the source, Vamifeport could provide a novel and effective alternative for managing this condition.
What evidence suggests that vamifeport might be an effective treatment for hemochromatosis?
Research has shown that vamifeport, an oral medication, can help control iron levels in the body. In studies with mice possessing a genetic mutation similar to humans with hereditary hemochromatosis, vamifeport significantly lowered blood iron levels and prevented liver iron buildup. This trial will test vamifeport at different dosages, with some participants receiving a low dose and others a high dose, to evaluate its effectiveness in managing iron overload in people with hereditary hemochromatosis. Additionally, vamifeport has improved blood cell production by reducing iron levels in the bloodstream, suggesting potential benefits for individuals with this condition.12678
Are You a Good Fit for This Trial?
This trial is for adults over 18 with hereditary hemochromatosis (HFE-HH), confirmed by medical history and specific MRI-based liver iron measurements. Participants must have certain levels of serum ferritin and transferrin saturation, as well as a body mass index within the specified range.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive vamifeport or placebo orally, twice daily up to Day 360
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Vamifeport
Trial Overview
The study tests Vamifeport against a placebo in people with HFE-HH to see if it can reduce liver iron concentration. It's a phase 2 trial where participants are randomly assigned to either the drug or placebo without knowing which one they're getting.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Placebo Group
Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
Participants will receive a high dose of vamifeport orally, BID up to Day 360.
Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.
Find a Clinic Near You
Who Is Running the Clinical Trial?
CSL Behring
Lead Sponsor
Dr. Paul McKenzie
CSL Behring
Chief Executive Officer since 2023
PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania
Dr. Bill Mezzanotte
CSL Behring
Chief Medical Officer since 2021
MD from Duke University
Citations
The oral ferroportin inhibitor vamifeport prevents liver iron ...
These data demonstrate that chronic vamifeport treatment significantly reduces serum iron levels and prevents liver iron loading in the Hfe C282Y mouse model ...
NCT07332091 | Efficacy and Safety of Vamifeport in Adult ...
For assessment of this outcome measure, data will be collected retrospectively from 1 year before baseline as well as during the study (up to Day 390). Up to ...
The oral ferroportin inhibitor vamifeport prevents liver iron ...
These data demonstrate that chronic vamifeport treatment significantly reduces serum iron levels and prevents liver iron loading in the Hfe C ...
Safety and pharmacodynamics of the ferroportin inhibitor ...
The ferroportin inhibitor, vamifeport, has been shown to improve erythropoiesis via decreases in serum iron and transferrin saturation levels in ...
5.
trial.medpath.com
trial.medpath.com/clinical-trial/956c2d7427f90cf7/nct07332091-vamifeport-hfe-hereditary-hemochromatosisEfficacy and Safety of Vamifeport in Adult Participants With ...
The primary objective of the study is to assess the effect of vamifeport treatment on magnetic resonance imaging (MRI)-based liver iron ...
Efficacy and Safety of Vamifeport in Adult Participants With ...
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis.
The oral ferroportin inhibitor vamifeport prevents liver iron ...
These data demonstrate that chronic vamifeport treatment significantly reduces serum iron levels and prevents liver iron loading in the Hfe C282Y mouse model ...
Efficacy and Safety of Vamifeport in Adult Participants W...
The primary objective of the study is to assess the effect of vamifeport treatment on magnetic resonance imaging (MRI)-based liver iron ...
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