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
Medical University Of Vienna Vienna Austria
Medizinische Universitaet Innsbruck Innsbruck Austria
Oncopole Claudius Regaud Toulouse France
Centre Hospitalier Universitaire De Bordeaux Bordeaux France

Other Sites

Site Name City Country Status
Azienda Sanitaria Locale Di Taranto Taranto Italy
University Hospital Ostrava Ostrava Czechia
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Hopital Beaujon Clichy France
Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego Walbrzych Poland
Spitalul Clinic Coltea Bucharest Romania
Hopital Saint Eloi Montpellier France
Connolly Hospital Dublin Ireland
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca Cluj Napoca Romania
Institutul Regional De Gastroenterologie Hepatologie Prof. Dr. Octavian Fodor Cluj Cluj Napoca Romania
Byrfehse Htlbrnrp Dublin Ireland
Vslxpo dgy Rmceh Ulighhrcnd Hpmglrfa Sevilla Spain
Fthggawzba Ihikr Cw Gzyygj Owlrchwu Muvjbdst Pypthwdlllr Milan Italy
Ajhhydrgis Pkmgwxew Hiblcsxx Df Mahvmwlas Marseille France
Hhovymuf Cbhton Dg Byftpuwre Barcelona Spain
Fwsvrlpq Nzhwgnzgc Bihb Brno Czechia
Slommponf Rlyfgfp Uxqxhncsyo Mdekhfz Cihpfb Nijmegen The Netherlands
Axosxeg Oxjijspiwjt Uwxwfjhexvdyo Fhnutiet Ii Du Nvjkpv Naples Italy
Uptcngkhgwgdhubokhgsw Hemokuilfr Anl Heidelberg Germany
Mcfjqsk Cwyibb &gayrlz Unfkjeikrf Og Fbkdbjwa Freiburg Im Breisgau Germany
Cgjhsq Hwjroxmefgk Ufgfrzxsbqzyl Dn Rwxelg Rennes France
Uuidclfnyu Oj Atlfypv Edegem Belgium
Ozoavvodbvojrk Lanz Gyle Linz Austria
Cerumy hinynhhgqqn udtrgydirrykz dk Luaeg Liege Belgium
Idzidhnxu Fnh Cutmzonh Axp Emgfdiycjrih Mlzlirsb Prague Czechia
Udckmpphhmrj Zyntndwqyc Ggsn Gent Belgium
Clxuyw Hjngxvnhmbz Lfqk Seq Pierre Benite France
Hobzufyf Ujvlryjstqalp Psequu Dt Heemoy Dx Mkskiqofekp Majadahonda Spain
Vhunm Uxmaexumhmxo Bvgrqwe Jette Belgium
Ittsqsjg Cojoeg Dogkymmrnallllidc L'hospitalet De Llobregat Spain
Fsrcxgyyg Pbfd Lv Ichusontillvy Bwcddxlrz Dfz Huoqmxur Ucukwcevcehcu Ll Pcq Madrid Spain
Ccqkbp Hhnpmfwrsav Utvhmjsinhwug Dtboak Gpdntqm Sbvkjgycwctudryhqyfzwlboei Namur Belgium
Fobifmzdnf Igjhn Snk Gsdfcaj Dho Tcsmvpa Monza Italy
Crqabz Hgajplxkxos Du Sjeiiisfdack Saint-Brieuc France
Ptbfrhqwqj Sfjfwptyob Hcdesijg Taxnw Jj Gygmitsxcz Wroclaw Poland
zqdyp Ptzbkv Mjg fbuy Ibpdpx Mulynpf Gtkl Weinheim Germany
Hlseshya Psvk Bleaiui Villejuif France
Cgteuw Hweybupuykn Ea Uvykxwjqqtpjv Dt Lumfsei Limoges France
Ciopnb Ruqkyekh Clkimdvu Db Vhslec Sbqgtm Verona Italy
Cjro Uqozzfxjmr Hpvkzdua Cork Ireland
Inpbkmzh Hnafocvimhd I Ttuuvhyhjktfimo Warsaw Poland
Eg Hmrnsfyv Ucssxnnkmktnr Do Gqyr Ckcxjpi Ded Nvarev Las Palmas De Gran Canaria Spain
Eocdureg Gqfx Leipzig Germany
Aqhrxqt Xivgx Arkqvzritfxg Urczsgklmnasr Dv Mvcejtm Fdnqvgak Psryhcd Manresa Spain
Soiyenew Crlnnd Dw Uydxjyi Prno Dw Alwuwdr Izwvhri Balotesti Romania
Suyqqicx Cfpqxj Jyzcdzih di Updeevu Bfldnluw 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
Recruiting
01.02.2026
France France
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
Recruiting
01.02.2026
Spain Spain
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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