NCT07332091

Brief 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). 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.

Trial Health

88
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
84

participants targeted

Target at P50-P75 for phase_2

Timeline
23mo left

Started Jan 2026

Geographic Reach
17 countries

93 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress13%
Jan 2026Apr 2028

First Submitted

Initial submission to the registry

January 8, 2026

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 12, 2026

Completed
10 days until next milestone

Study Start

First participant enrolled

January 22, 2026

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 6, 2028

Expected
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 6, 2028

Last Updated

March 27, 2026

Status Verified

March 1, 2026

Enrollment Period

2.1 years

First QC Date

January 8, 2026

Last Update Submit

March 25, 2026

Conditions

Keywords

Small Molecule Ferroportin InhibitorHereditary hemochromatosisIron overload

Outcome Measures

Primary Outcomes (1)

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

    At Baseline and Day 360

Secondary Outcomes (27)

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

    Up to Day 390

  • Percentage of participants with TEAEs

    Up to Day 390

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

    Up to Day 390

  • Percentage of participants with treatment-emergent SAEs

    Up to Day 390

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

    From Baseline to Day 390

  • +22 more secondary outcomes

Study Arms (3)

Vamifeport Low Dose

EXPERIMENTAL

Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.

Drug: Vamifeport

Vamifeport High Dose

EXPERIMENTAL

Participants will receive a high dose of vamifeport orally, BID up to Day 360.

Drug: Vamifeport

Placebo

PLACEBO COMPARATOR

Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.

Drug: Placebo

Interventions

Vamifeport capsule administered orally.

Vamifeport High DoseVamifeport Low Dose

Placebo capsule matching IP administered orally.

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 3 and 16 mg/g (53.7 and 286.5 millimol per kilogram \[mmol/kg\]) dry weight (dw) at Screening.
  • Body mass index between 18.5 and 32 kilograms per meter squared (kg/m\^2).

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (96)

Banner MD Anderson

Gilbert, Arizona, 85234-2165, United States

NOT YET RECRUITING

Infinity Clinical Trials

San Diego, California, 92108, United States

NOT YET RECRUITING

Medical Oncology Associates of San Diego

San Diego, California, 92123, United States

NOT YET RECRUITING

Green Leaf Clinical Trials

Jacksonville, Florida, 32258, United States

RECRUITING

Indiana University Health University Hospital

Indianapolis, Indiana, 46202-5149, United States

NOT YET RECRUITING

Ochsner Medical Complex - High Grove

Baton Rouge, Louisiana, 70836, United States

NOT YET RECRUITING

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

NOT YET RECRUITING

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

Bethesda, Maryland, 20817, United States

NOT YET RECRUITING

James M. Stockman Cancer Institute

Frederick, Maryland, 21702-4337, United States

NOT YET RECRUITING

University of Michigan Health System (UMHS)

Ann Arbor, Michigan, 48109, United States

NOT YET RECRUITING

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

Duluth, Minnesota, 55805, United States

NOT YET RECRUITING

Hunterdon Hematology Oncology, LLC

Flemington, New Jersey, 08822, United States

NOT YET RECRUITING

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27517-7518, United States

NOT YET RECRUITING

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

Durham, North Carolina, 27710-4000, United States

NOT YET RECRUITING

Hightower Clinical - Oklahoma Cancer Center

Oklahoma City, Oklahoma, 73174, United States

NOT YET RECRUITING

Intermountain Medical Center

Murray, Utah, 84107, United States

NOT YET RECRUITING

Washington State Univ Elson S. Floyd College of Medicine

Spokane, Washington, 99202-2131, United States

RECRUITING

Royal Brisbane and Women's Hospital

Brisbane, Australia

NOT YET RECRUITING

Gallipoli Medical Research

Chandler, Australia

NOT YET RECRUITING

Monash Medical Centre

Clayton, Australia

NOT YET RECRUITING

Trials West

Perth, Australia

NOT YET RECRUITING

Westmead Hospital for Medical Research

Westmead, 2145, Australia

NOT YET RECRUITING

Medical University of Innsbruck

Innsbruck, Austria

NOT YET RECRUITING

Ordensklinikum Linz - Barmherzige Schwestern

Linz, Austria

NOT YET RECRUITING

Medical University Vienna

Vienna, Austria

NOT YET RECRUITING

Universitair Ziekenhuis Antwerpen (UZA)

Edegem, Belgium

NOT YET RECRUITING

Ghent University Hospital

Ghent, Belgium

NOT YET RECRUITING

UZ Brussel

Jette, Belgium

NOT YET RECRUITING

Centre Hospitalier Universitaire de Liège (CHU de Liège)

Liège, Belgium

NOT YET RECRUITING

CHU UCL Namur - Site Godinne

Yvoir, Belgium

NOT YET RECRUITING

Libin Cardiovascular Institute University of Calgary

Calgary, Canada

NOT YET RECRUITING

McMaster University-St. Josephs Healthcare Hamilton

Hamilton, Canada

NOT YET RECRUITING

University of Manitoba

Winnipeg, Canada

NOT YET RECRUITING

Fakultní Nemocnice Brno

Brno, Czechia

NOT YET RECRUITING

Fakultni nemocnice Ostrava

Ostrava, Czechia

NOT YET RECRUITING

Institut Klinicke a Experimentalni Mediciny

Prague, Czechia

NOT YET RECRUITING

Aarhus University Hospital

Aarhus, Denmark

NOT YET RECRUITING

Bispebjerg Hospital

Copenhagen, Denmark

NOT YET RECRUITING

Copenhagen University Hospital - Hvidovre

Hvidovre, Denmark

NOT YET RECRUITING

Aphp Avicenne

Bobigny, France

NOT YET RECRUITING

Chu Dupuytren

Limoges, France

NOT YET RECRUITING

CRMR Maladies du Globule Rouge, Hôpital de la Timone

Marseille, France

NOT YET RECRUITING

CHU de Montpellier- Hôpital Saint Eloi

Montpellier, France

NOT YET RECRUITING

GHRMSA

Mulhouse, France

NOT YET RECRUITING

CHU de Bordeaux - Hôpital Haut Leveque

Pessac, France

NOT YET RECRUITING

Centre Hospitalier Lyon Sud/Hospices Civils de Lyon

Pierre-Bénite, France

NOT YET RECRUITING

Chu Rennes

Rennes, France

NOT YET RECRUITING

Centre Hospitalier de Saint Brieuc

Saint-Brieuc, France

NOT YET RECRUITING

CHU Toulouse

Toulouse, France

NOT YET RECRUITING

Hôpital Paul Brousse

Villejuif, France

NOT YET RECRUITING

Universitätsklinikum Freiburg

Freiburg im Breisgau, Germany

NOT YET RECRUITING

University Hospital Heidelberg

Heidelberg, Germany

NOT YET RECRUITING

EUGASTRO GmbH

Leipzig, Germany

NOT YET RECRUITING

MVZ für Innere Medizin Weinheim

Weinheim, Germany

NOT YET RECRUITING

Cork University Hospital

Cork, Ireland

NOT YET RECRUITING

Beaumont Hospital

Dublin, Ireland

NOT YET RECRUITING

Connolly Hospital Blanchardstown

Dublin, Ireland

NOT YET RECRUITING

ASL Brindisi - Presidio Ospedaliero Di Summa - Perrino

Brindisi, Italy

NOT YET RECRUITING

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, Italy

NOT YET RECRUITING

University Hospital of Modena

Modena, Italy

NOT YET RECRUITING

Fondazione IRCCS San Gerardo dei Tintori

Monza, Italy

NOT YET RECRUITING

AORN Cardarelli

Naples, Italy

NOT YET RECRUITING

University of Verona - Azienda Ospedaliera Universitaria Integrata Verona

Verona, Italy

NOT YET RECRUITING

Maastricht UMC

Maastricht, Netherlands

NOT YET RECRUITING

Radboud UMC

Nijmegen, Netherlands

NOT YET RECRUITING

Auckland City Hospital

Auckland, 1023, New Zealand

NOT YET RECRUITING

Aotearoa Clinical Trials Trust- Middlemore Hospital

Auckland, 2025, New Zealand

NOT YET RECRUITING

Instytut Hematologii i Transfuzjologii

Warsaw, Poland

NOT YET RECRUITING

Specjalistyczny Szpital im. Alfreda Sokolowskiego

Wałbrzych, Poland

NOT YET RECRUITING

Wojewodzki Szpital im. J Gromkowskiego we Wroclawiu

Wroclaw, Poland

NOT YET RECRUITING

Spitalul Clinic de Urgenta Prof Dr Agrippa Ionescu-Balotesti

Baloteşti, Romania

NOT YET RECRUITING

Bistrița County Emergency Clinical Hospital

Bistriţa, Romania

NOT YET RECRUITING

Coltea Clinical Hospital

Bucharest, Romania

NOT YET RECRUITING

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

Cluj-Napoca, Romania

NOT YET RECRUITING

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

Cluj-Napoca, Romania

NOT YET RECRUITING

Hospital Universitari Germans Trias i Pujol

Badalona, Spain

NOT YET RECRUITING

Hospital Clinic Barcelona

Barcelona, Spain

NOT YET RECRUITING

HUGC Doctor Negrin

LAS Palmas de GC, Spain

NOT YET RECRUITING

Hospital Universitario La Paz

Madrid, Spain

NOT YET RECRUITING

Hospital Universitario Puerta de Hierro - Majadahonda

Majadahonda, Spain

NOT YET RECRUITING

Althaia Foundation. Hospital Sant Joan de Deu de Manresa

Manresa, Spain

NOT YET RECRUITING

Hospital Universitario Virgen del Rocío

Seville, Spain

NOT YET RECRUITING

University Hospital Inselspital Bern

Bern, Switzerland

NOT YET RECRUITING

Centre Hospitalier Universitaire Vaudois

Lausanne, Switzerland

NOT YET RECRUITING

Epatocentro Ticino

Lugano, Switzerland

NOT YET RECRUITING

Royal Victoria Hospital

Belfast, United Kingdom

NOT YET RECRUITING

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

Bristol, United Kingdom

NOT YET RECRUITING

Royal Liverpool University Hospital

Liverpool, United Kingdom

NOT YET RECRUITING

King's College Hospital

London, United Kingdom

NOT YET RECRUITING

St Thomas Hospital

London, United Kingdom

NOT YET RECRUITING

Norfolk and Norwich University Hospital

Norwich, United Kingdom

NOT YET RECRUITING

Nottingham University Hospitals City Campus

Nottingham, United Kingdom

NOT YET RECRUITING

John Radcliffe Hospital - Oxford University Hospitals NHS

Oxford, United Kingdom

NOT YET RECRUITING

Derriford Hospital

Plymouth, United Kingdom

NOT YET RECRUITING

University Hospital Southampton NHS Foundation Trust

Southampton, United Kingdom

NOT YET RECRUITING

South Warwickshire University Foundation Trust

Warwick, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

HemochromatosisIron Overload

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesIron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Trial Registration Coordinator

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: This is multicenter, randomized, placebo-controlled double-blind, parallel-group study.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 8, 2026

First Posted

January 12, 2026

Study Start

January 22, 2026

Primary Completion (Estimated)

March 6, 2028

Study Completion (Estimated)

April 6, 2028

Last Updated

March 27, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
Access Criteria
Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Locations