RecruitingPHASE2INTERVENTIONAL

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

This study is looking into a new medicine called Vamifeport for adults who have a common genetic condition called hereditary hemochromatosis (HFE-HH). This condition causes too much iron to build up in the body, particularly in the liver. The main goal of the study is to find out if Vamifeport can help lower the iron levels in the liver, which will be measured using a special type of MRI scan. To get a clear picture, some participants will receive the new medicine, while others will receive a dummy pill (placebo). This helps researchers understand the true effect of Vamifeport compared to not having the active medicine. The study is in an early stage, called Phase 2, and will involve several medical checks.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

CSL Behring

Enrolment target

Start

22 Jan 2026

Estimated completion

06 Apr 2028

Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

What is this study about?

This study is exploring a new medicine called Vamifeport for adults who have a condition known as hereditary hemochromatosis (HFE-HH). This is a genetic condition meaning it's passed down in families, and it causes the body to absorb too much iron from food. Over time, this extra iron can build up in organs like the liver, which can cause health problems if not managed.

The main purpose of this study is to see if Vamifeport can safely and effectively reduce the amount of iron stored in the liver. Researchers will use a special type of MRI scan to measure iron levels in the liver before and after treatment. They want to understand how well this new medicine works compared to a 'dummy' pill that contains no active medicine, which is called a placebo. By comparing these two groups, scientists can be more sure about the effects of Vamifeport itself.

This is a Phase 2 study, which means it's an early but important step in developing new medicines. It helps researchers gather more information about how the medicine works, what a good dose might be, and checks for any side effects. The results will help decide if this medicine should be tested in larger studies in the future.

Key takeaways

This study tests a new medicine (Vamifeport) for iron overload in hereditary hemochromatosis.
It aims to reduce liver iron levels, measured by special MRI scans.
You might receive the new medicine or a dummy pill (placebo).
Regular health checks, blood tests, and scans are part of participating.
It's a Phase 2 study, an early step in medicine development.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to be an adult, aged 18 or over, and have signed a form saying you understand the study and agree to take part. A doctor must have already diagnosed you with hereditary hemochromatosis (HFE-HH) in the past. We'll also need to see that you have too much iron in your body currently. This will be checked by blood tests showing specific iron levels and a special MRI scan of your liver showing iron buildup. Your body weight and height (BMI) also need to be within a certain healthy range.

There are also some reasons why you might not be able to join. For example, if you have other serious health problems, unusual blood test results, or heart issues that doctors think are important, you might not be eligible. This is to make sure the study is as safe as possible for everyone involved.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult (18 years or older)?
Have you been diagnosed with hereditary hemochromatosis (HFE-HH)?
Do your doctors think you have too much iron in your body currently?
Do you have a healthy body weight for your height?
Do you have any serious ongoing health problems that might make you ineligible?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be randomly assigned to one of two groups: one receiving the new medicine, Vamifeport, and the other receiving a dummy pill (placebo). Neither you nor your study doctor will know which you are receiving, which helps make the study fair. You'll take the study medicine or placebo regularly as instructed.

Throughout the study, you'll have several visits to the clinic. These visits will involve various health checks, blood tests, and liver MRI scans to measure your iron levels. There will also be appointments to check on your overall health and look for any changes or side effects. The total duration of your participation in the study will involve initial checks, the period of taking the study medicine, and a follow-up period after you stop taking it.

Potential risks and benefits

Taking part in this study could potentially help reduce the iron overload in your body, which might improve your health if the new medicine, Vamifeport, proves effective. However, as with any new medicine, there's always a possibility of side effects or unknown risks. The study team will carefully monitor your health throughout. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (98)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Banner MD Anderson
Verified postcode
Gilbert, United States· Not yet recruiting
Infinity Clinical Trials
Verified postcode
San Diego, United States· Not yet recruiting
Medical Oncology Associates of San Diego
Verified postcode
San Diego, United States· Not yet recruiting
Green Leaf Clinical Trials
Verified postcode
Jacksonville, United States· Recruiting
Indiana University Health University Hospital
Verified postcode
Indianapolis, United States· Recruiting
Ochsner Medical Complex - High Grove
Verified postcode
Baton Rouge, United States· Not yet recruiting
Johns Hopkins University School of Medicine
Verified postcode
Baltimore, United States· Not yet recruiting
American Oncology Partners, PA dba The Center for Cancer and Blood Disorders
Verified postcode
Bethesda, United States· Recruiting
James M. Stockman Cancer Institute
Verified postcode
Frederick, United States· Not yet recruiting
University of Michigan Health System (UMHS)
Verified postcode
Ann Arbor, United States· Not yet recruiting
Aspirus St. Luke's Clinic - Duluth - Oncology & Hematology
Verified postcode
Duluth, United States· Recruiting
Hunterdon Hematology Oncology, LLC
Verified postcode
Flemington, United States· Not yet recruiting

Common questions

What is hereditary hemochromatosis (HFE-HH)?

It's a genetic condition where your body absorbs and stores too much iron, which can build up in organs like the liver and cause health problems.

What is a 'dummy pill' or placebo?

A dummy pill looks exactly like the study medicine but doesn't contain any active ingredients. It helps researchers understand the true effect of the medicine being tested.

What does a liver MRI scan involve?

It's a special type of scan that uses strong magnets and radio waves to create detailed pictures of your liver, allowing doctors to precisely measure iron levels there.

Why is this called a 'Phase 2' study?

Phase 2 studies are early stages of drug development. They aim to see if a new medicine works, find a good dose, and check for safety in a larger group of people than initial tests.

Will I know if I'm getting the real medicine or the placebo?

No, during the study, neither you nor your study doctor will know whether you are receiving Vamifeport or the placebo. This is to ensure the study results are unbiased.

How to find out more

Trial Registration Coordinator

clinicaltrials@cslbehring.com +16108784697 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.