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

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What is this study about?

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.

Who Can Join the Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Universitaet Innsbruck Innsbruck Austria
Medical University Of Vienna Vienna Austria
Azienda Ospedaliera Universitaria Federico II Di Napoli Naples Italy
Universitaetsklinikum Heidelberg AöR Heidelberg Germany
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Oncopole Claudius Regaud Toulouse France

Other Sites

Site Name City Country Status
Centro Ricerche Cliniche Di Verona S.r.l. Verona Italy
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Centre hospitalier universitaire de Liege Liege Belgium
Universitair Ziekenhuis Gent Gent Belgium
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Institutul Regional De Gastroenterologie Hepatologie Prof. Dr. Octavian Fodor Cluj Cluj Napoca Romania
Instytut Hematologii I Transfuzjologii Warsaw Poland
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
El Hospital Universitario De Gran Canaria Dr. Negrin Las Palmas De Gran Canaria Spain
Eugastro GmbH Leipzig Germany
Althaia Xarxa Assistencial Universitaria De Manresa Fundacio Privada Manresa Spain
Spitalul Clinic De Urgenta Prof Dr Agrippa Ionescu Balotesti Romania
Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego Walbrzych Poland
Azienda Sanitaria Locale Di Taranto Taranto Italy
Spitalul Clinic Coltea Bucharest Romania
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Clinic De Barcelona Barcelona Spain
Fakultni Nemocnice Brno Brno Czechia
Stichting Radboud University Medical Center Nijmegen The Netherlands
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca Cluj Napoca Romania
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Centre Hospitalier Universitaire De Rennes Rennes France
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Vrije Universiteit Brussel Jette Belgium
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur Namur Belgium
Hospital Paul Brousse Villejuif France
Hopital Saint Eloi Montpellier France
University Hospital Ostrava Ostrava Czechia
Connolly Hospital Dublin Ireland
Beaumont Hospital Dublin Ireland
Ogliteejrqrmwz Lied Gbui Linz Austria
Isxaoukez Fut Cgqmvddg Ank Ebxcjpakuqxo Mtcqrnyf Prague Czechia
Cgkv Uwqwqjvlyu Hopuewvj Cork Ireland
zrgfi Pswnlp Mws frqw Imwzho Myrhtzp Gqel Weinheim Germany
Pkbtmweuwd Snnvcrclsy Hygcajyj Tplyb Jo Gwuzvugols Wroclaw Poland
Cbkqck Hgxogttvave Dl Sqvcegafpjip Saint-Brieuc France
Cmdpqv Htysugexmgy Eo Uyjrfhiwlodvj Dm Lzteqpe Limoges France
Absrxlrnff Phuzjapi Hncevieb Dm Mygzbgpcg Marseille France
Unbmkmtxgf Os Aaspmhx Edegem Belgium
Iwfizpeu Cgpizc Dvargvxndyaxndbcc L'hospitalet De Llobregat Spain
Fvzhvfhqi Pggu Lt Iavtlnouyvvde Brgsutjtf Dsc Htynscfs Uhsvddtfkbcbh Lv Plc Madrid Spain
Slxbswub Chgjud Jsyeehap dw Ukldyva Befdlatq Bistrița Romania

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not yet recruiting
01.02.2026
Belgium Belgium
Not yet recruiting
01.02.2026
Czechia Czechia
Not yet recruiting
01.02.2026
France France
Not yet recruiting
01.02.2026
Germany Germany
Not yet recruiting
01.02.2026
Ireland Ireland
Not yet recruiting
01.02.2026
Italy Italy
Not yet recruiting
01.02.2026
Poland Poland
Not yet recruiting
01.02.2026
Romania Romania
Not yet recruiting
01.02.2026
Spain Spain
Not yet recruiting
01.02.2026
The Netherlands The Netherlands
Not yet recruiting
01.02.2026

Trial locations

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.

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 ID:
2025-523793-16-00
Protocol code:
CSL624_2001
Trial Phase:
Therapeutic exploratory (Phase II)

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