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Homeostatic Iron Regulator Gene-related Hereditary HemochromatosisPhase 2NCT07332091

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

A Phase 2 study testing Vamifeport in people with Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis, run by CSL Behring at 100 sites in 18 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary hemochromatosis (HFE-HH). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Vamifeport
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
84 participants
Where
100 sitesin 18 countries
Study dates
22 Jan 2026to 15 May 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adult (≥ 18 years) and has provided written informed consent.
  • Confirmed diagnosis of HFE-HH in medical history.
  • Evidence of iron overload as shown by:
  • TSAT > 45% (confirmed at 2 visits, at least 14 days apart) at Screening; and
  • Serum ferritin ≥ 200 nanogram per milliliter (ng/mL) and < 5000 ng/mL (confirmed at 2 visits, at least 14 days apart) at Screening; and
  • MRI-based LIC between 2.4 and 16 mg/g (43.0 and 286.5 mmol/kg) dry weight (dw) at Screening.
  • Body mass index between 18.5 and 34.9 kilograms per meter squared (kg/m^2).

Who cannot join

  • Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.

Trial locations and contacts

100 sites in 18 countries.

100 sites in 18 countries

United States20 sites

  • University of Michigan Health System (UMHS)

    Ann Arbor, Michigan, 48109

  • Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21205

  • Ochsner Medical Complex - High Grove

    Baton Rouge, Louisiana, 70836

  • American Oncology Partners, PA dba The Center for Cancer and Blood Disorders

    Bethesda, Maryland, 20817

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27517-7518

  • Aspirus St. Luke's Clinic - Duluth - Oncology & Hematology

    Duluth, Minnesota, 55805

  • Duke University Medical Center (Duke South Clinics) -40 Duke Medicine Cir

    Durham, North Carolina, 27710-4000

  • Hunterdon Hematology Oncology, LLC

    Flemington, New Jersey, 08822

  • James M. Stockman Cancer Institute

    Frederick, Maryland, 21702-4337

  • Banner MD Anderson

    Gilbert, Arizona, 85234-2165

  • Indiana University Health University Hospital

    Indianapolis, Indiana, 46202-5149

  • Green Leaf Clinical Trials

    Jacksonville, Florida, 32258

  • Global Medical Research Management LLC

    Miami, Florida, 33177

  • Intermountain Medical Center

    Murray, Utah, 84107

  • Hightower Clinical - Oklahoma Cancer Center

    Oklahoma City, Oklahoma, 73174

  • Infinity Clinical Trials

    San Diego, California, 92108

  • Medical Oncology Associates of San Diego

    San Diego, California, 92123

  • Velocity Clinical Research - Seattle

    Seattle, Washington, 98105

  • Washington State Univ Elson S. Floyd College of Medicine

    Spokane, Washington, 99202-2131

  • Cancer Care Associates of York

    York, Pennsylvania, 17403-5060

Other trials in United States

United Kingdom12 sites

  • Royal Victoria Hospital

    Belfast

  • University Hospitals Bristol and Weston NHS trust, Bristol Haematology and Oncology Centre

    Bristol

  • University Hospital Hairmyres-PPDS

    Glasgow, Glasgow City Region, SE5 9RS

  • Royal Liverpool University Hospital

    Liverpool

  • King's College Hospital

    London

  • St Thomas Hospital

    London

  • Norfolk and Norwich University Hospital

    Norwich

  • Nottingham University Hospitals City Campus

    Nottingham

  • John Radcliffe Hospital - Oxford University Hospitals NHS

    Oxford

  • Derriford Hospital

    Plymouth

  • University Hospital Southampton NHS Foundation Trust

    Southampton

  • South Warwickshire University Foundation Trust

    Warwick

Other trials in United Kingdom

France11 sites

  • Aphp Avicenne

    Bobigny, Île-de-France Region, 93000

  • Chu Dupuytren

    Limoges

  • CRMR Maladies du Globule Rouge, Hôpital de la Timone

    Marseille

  • CHU de Montpellier- Hôpital Saint Eloi

    Montpellier

  • GHRMSA

    Mulhouse

  • CHU de Bordeaux - Hôpital Haut Leveque

    Pessac

  • Centre Hospitalier Lyon Sud/Hospices Civils de Lyon

    Pierre-Bénite

  • Chu Rennes

    Rennes

  • Centre Hospitalier de Saint Brieuc

    Saint-Brieuc

  • CHU Toulouse

    Toulouse

  • Hôpital Paul Brousse

    Villejuif

Other trials in France

Spain7 sites

  • Hospital Universitari Germans Trias i Pujol

    Badalona

  • Hospital Clinic Barcelona

    Barcelona

  • HUGC Doctor Negrin

    LAS Palmas de GC

  • Hospital Universitario La Paz

    Madrid, 28046

  • Hospital Universitario Puerta de Hierro - Majadahonda

    Majadahonda

  • Althaia Foundation. Hospital Sant Joan de Deu de Manresa

    Manresa

  • Hospital Universitario Virgen del Rocío

    Seville

Other trials in Spain

Italy6 sites

  • ASL Brindisi - Presidio Ospedaliero Di Summa - Perrino

    Brindisi

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

    Milan

  • University Hospital of Modena

    Modena

  • Fondazione IRCCS San Gerardo dei Tintori

    Monza

  • AORN Cardarelli

    Naples

  • University of Verona - Azienda Ospedaliera Universitaria Integrata Verona

    Verona

Other trials in Italy

Australia5 sites

  • Royal Brisbane and Women's Hospital

    Brisbane, 4006

  • Gallipoli Medical Research

    Chandler, Queensland, 4120

  • Monash Medical Centre

    Clayton

  • Trials West

    Perth

  • Westmead Hospital for Medical Research

    Westmead, 2145

Other trials in Australia

Belgium5 sites

  • Universitair Ziekenhuis Antwerpen (UZA)

    Edegem, 2650

  • Ghent University Hospital

    Ghent, West Vlaanderen, 9000

  • UZ Brussel

    Jette

  • Centre Hospitalier Universitaire de Liège (CHU de Liège)

    Liège

  • CHU UCL Namur - Site Godinne

    Yvoir

Other trials in Belgium

Romania5 sites

  • Spitalul Clinic de Urgenta Prof Dr Agrippa Ionescu-Balotesti

    Baloteşti

  • Bistrița County Emergency Clinical Hospital

    Bistriţa

  • Coltea Clinical Hospital

    Bucharest

  • L'Institute Oncologique Prof. Dr. Ion Chiricuta (IOCN)

    Cluj-Napoca

  • Prof. Dr.Octavian Fodor Regional Institute of Gastroenterology-Hepatology

    Cluj-Napoca

Other trials in Romania

Germany4 sites

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau

  • University Hospital Heidelberg

    Heidelberg

  • EUGASTRO GmbH

    Leipzig

  • MVZ für Innere Medizin Weinheim

    Weinheim

Other trials in Germany

Austria3 sites

  • Medical University of Innsbruck

    Innsbruck

  • Ordensklinikum Linz - Barmherzige Schwestern

    Linz

  • Medical University Vienna

    Vienna

Other trials in Austria

Canada3 sites

  • Libin Cardiovascular Institute University of Calgary

    Calgary

  • McMaster University-St. Josephs Healthcare Hamilton

    Hamilton

  • University of Manitoba

    Winnipeg

Other trials in Canada

Czechia3 sites

  • Fakultní Nemocnice Brno

    Brno

  • Fakultni nemocnice Ostrava

    Ostrava

  • Institut Klinicke a Experimentalni Mediciny

    Prague

Other trials in Czechia

Denmark3 sites

  • Aarhus University Hospital

    Aarhus

  • Bispebjerg Hospital

    Copenhagen

  • Copenhagen University Hospital - Hvidovre

    Hvidovre

Other trials in Denmark

Ireland3 sites

  • Cork University Hospital

    Cork

  • Connolly Hospital Blanchardstown

    Dublin, D15X40D

  • Beaumont Hospital

    Dublin

Other trials in Ireland

Poland3 sites

  • Specjalistyczny Szpital im. Alfreda Sokolowskiego

    Wałbrzych

  • Instytut Hematologii i Transfuzjologii

    Warsaw

  • Wojewodzki Szpital im. J Gromkowskiego we Wroclawiu

    Wroclaw

Other trials in Poland

Switzerland3 sites

  • University Hospital Inselspital Bern

    Bern

  • Centre Hospitalier Universitaire Vaudois

    Lausanne

  • Epatocentro Ticino

    Lugano

Other trials in Switzerland

Netherlands2 sites

  • Maastricht UMC

    Maastricht

  • Radboud UMC

    Nijmegen

Other trials in Netherlands

New Zealand2 sites

  • Auckland City Hospital

    Auckland, 1023

  • Aotearoa Clinical Trials Trust- Middlemore Hospital

    Auckland, 2025

Other trials in New Zealand

About this study

Official title: A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study of the Efficacy and Safety of Vamifeport in Adult Subjects With HFE-related Hereditary Hemochromatosis (FERROCLEAR Study)

This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary hemochromatosis (HFE-HH). The primary objective of the study is to assess the effect of vamifeport treatment on magnetic resonance imaging (MRI)-based liver iron concentration (LIC) in adult participants with HFE-HH.

Being tested
Vamifeport, Placebo
Keywords
Small Molecule Ferroportin Inhibitor, Hereditary hemochromatosis, Iron overload
Medical subject headings
Hemochromatosis, Iron Overload

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VamifeportDrug

    Vamifeport capsule administered orally.

  • PlaceboDrug

    Placebo capsule matching IP administered orally.

Study groups in NCT07332091
Study groupRoleWhat they receive
Vamifeport Low DoseExperimentalParticipants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
Receives: Drug: Vamifeport
Vamifeport High DoseExperimentalParticipants will receive a high dose of vamifeport orally, BID up to Day 360.
Receives: Drug: Vamifeport
PlaceboPlacebo comparatorParticipants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)

    Measured: At Baseline and Day 360

Secondary outcomes (27)

  1. Number of participants with treatment-emergent adverse events (TEAEs)

    Measured: Up to Day 390

  2. Percentage of participants with TEAEs

    Measured: Up to Day 390

  3. Number of participants with treatment-emergent serious adverse events (SAEs)

    Measured: Up to Day 390

  4. Percentage of participants with treatment-emergent SAEs

    Measured: Up to Day 390

  5. Number of participants with clinically significant change from baseline in clinical safety laboratory tests and 12-lead electrocardiogram (ECG)

    Clinical safety laboratory tests will include hematology, biochemistry and coagulation.

    Measured: From Baseline to Day 390

  6. Percentage of participants with clinically significant change from baseline in clinical safety laboratory tests and 12-lead ECG

    Clinical safety laboratory tests will include hematology, biochemistry and coagulation.

    Measured: From Baseline to Day 390

  7. Change from baseline in transferrin saturation (TSAT) (measured at trough)

    Measured: From Baseline to Day 360

  8. Number of participants with TSAT less than or equal to (<=) 45% (measured at trough)

    Measured: From Baseline to Day 360

  9. Percentage of participants with TSAT <= 45% (measured at trough)

    Measured: From Baseline to Day 360

  10. Number of participants with 25% reduction in MRI-based LIC

    Measured: At Day 180 and 360

  11. Number of participants with 50% reduction in MRI-based LIC

    Measured: At Day 180 and Day 360

  12. Number of participants with TSAT ≤ 45% or MRI-based LIC < 5 milligrams per gram (mg/g)

    Measured: At Day 360

  13. Number of participants with TSAT ≤ 45% or MRI-based LIC < 2 mg/g

    Measured: At Day 360

  14. Change from baseline in joint pain score in a visual analog scale (VAS)

    The VAS is a single-item questionnaire. Participants will be asked to record their joint pain at its worst over the previous week, from 0 (No pain) to 10 (Worst possible pain).

    Measured: From Baseline to Day 360

  15. Change from baseline in modified fatigue impact scale (MFIS) total score

    Participants will be asked to read a list of 21 statements and assess how often fatigue has affected them in terms of physical, cognitive, and psychosocial functioning, over the previous 4 weeks, using a 5-point scale from 0 (Never) to 4 (Almost always). The total score is obtained by summing the scores of all the 21 items. Higher scores indicate a greater impact of fatigue on a participant's activities.

    Measured: From Baseline to Day 360

  16. Change from baseline in health-related quality of life: EuroQoL 5-dimension 5-level instrument (EQ-5D-5L)

    The EQ-5D-5L is a standardized measure of health status that provides a simple, generic measure of health for clinical and economic appraisal. Participants will complete the questionnaire based on their health on that day. The EQ-5D-5L descriptive profile includes 5 dimensions: mobility, self-care, usual activities, pain / discomfort, and anxiety / depression. Participants will rate each dimension based on 5 levels of severity: no problems, slight problems, moderate problems, severe problems, and extreme problems. A higher score indicates a more severe outcome than a lower score.

    Measured: From Baseline to Days 180, 360, and 390

  17. Change from baseline in health-related quality of life: VAS (Arthralgia)

    The VAS is a single-item questionnaire. Participants will be asked to record their joint pain at its worst over the previous week, from 0 (No pain) to 10 (Worst possible pain).

    Measured: From Baseline to Day 180, 360, and 390

  18. Change from baseline in health-related quality of life: Patient global impression of change in clinical status

    The PGI - Change (PGI-C) instrument is a self-reported measure that reflects belief about the efficacy of treatment. Participants will be asked to rate the change in their overall symptoms since they started taking investigational product (IP) on a 5-point scale, ranging from 1 (Much improved) to 5 (Much worse). A lower score reflects an improvement in clinical status.

    Measured: From Baseline to Day 180, 360, and 390

  19. Change from baseline in health-related quality of life: Patient global impression of severity

    The PGI - Severity (PGI-S) instrument is a self-reported questionnaire and consists of 1 item that measures disease severity. Participants will be asked to rate the severity of their overall symptoms over the previous week on a 5-point scale from 1 (None) to 5 (Very severe). A higher score reflects a more severe outcome than a lower score.

    Measured: From Baseline to Day 180, 360, and 390

  20. Change from baseline in health-related quality of life: MFIS physical, cognitive, and psychosocial subscales

    Participants will be asked to read a list of 21 statements and assess how often fatigue has affected them in terms of physical, cognitive, and psychosocial functioning, over the previous 4 weeks, using a 5-point scale from 0 (Never) to 4 (Almost always).

    Items on the MFIS will be aggregated into 3 subscales (Physical, Cognitive, or Psychosocial). Higher scores indicate a greater impact of fatigue on a participant's activities.

    Measured: From Baseline to Day 180, 360, and 390

  21. Vamifeport plasma concentrations after a single dose

    Measured: At Day 1

  22. Vamifeport plasma concentrations at steady state

    Measured: At Days 15, 180, and 360

  23. Change from baseline in total serum iron (measured at trough)

    Measured: From Baseline to Day 360

  24. Change from baseline in serum ferritin (measured at trough)

    Measured: From Baseline to Day 360

  25. Frequency of rescue therapy use

    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).

    Measured: Up to Day 390

  26. Duration of rescue therapy use

    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).

    Measured: Up to Day 390

  27. Time to first use of rescue therapy

    Measured: Up to Day 360

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jan 2026
  2. Study start22 Jan 2026
  3. Primary completion15 Apr 2028 (estimated)
  4. Study completion15 May 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This is multicenter, randomized, placebo-controlled double-blind, parallel-group study.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
84 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07332091
Sponsor's protocol ID
CSL624_2001
Other IDs
2025-523793-16-00, 2025
Status last verified
Oct 2026
First submitted
8 Jan 2026
Last updated
21 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Vamifeport in people with Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis, run by CSL Behring at 100 sites in 18 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 100 sites in United States, United Kingdom, France, Spain, Italy and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC) (time frame: At Baseline and Day 360). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07332091.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2026.

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