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

NCT07332091 · tracked via the Priya Life Science Ireland tracker

Sponsor

CSL Behring

Phase

Phase 2

Started

2026-01-22

Last updated

2026-09-16

Condition(s) studied

Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

Investigational drug(s) / intervention(s)

VamifeportPlacebo

Vamifeport: Vamifeport capsule administered orally.

Placebo: Placebo capsule matching IP administered orally.

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * 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). Exclusion Criteria: * Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.

Primary outcome measure(s)

Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC) — At Baseline and Day 360

Trial sites (100)

Facility	City	Region	Status
Banner MD Anderson	Gilbert	Arizona	Not Yet Recruiting
Infinity Clinical Trials	San Diego	California	Not Yet Recruiting
Medical Oncology Associates of San Diego	San Diego	California	Not Yet Recruiting
Green Leaf Clinical Trials	Jacksonville	Florida	Recruiting
Global Medical Research Management	Plantation	Florida	Recruiting
Indiana University Health University Hospital	Indianapolis	Indiana	Recruiting
Ochsner Medical Complex - High Grove	Baton Rouge	Louisiana	Recruiting
Johns Hopkins University School of Medicine	Baltimore	Maryland	Not Yet Recruiting
American Oncology Partners, PA dba The Center for Cancer and Blood Disorders	Bethesda	Maryland	Recruiting
James M. Stockman Cancer Institute	Frederick	Maryland	Recruiting
University of Michigan Health System (UMHS)	Ann Arbor	Michigan	Recruiting
Aspirus St. Luke's Clinic - Duluth - Oncology & Hematology	Duluth	Minnesota	Recruiting
Hunterdon Hematology Oncology, LLC	Flemington	New Jersey	Not Yet Recruiting
University of North Carolina at Chapel Hill	Chapel Hill	North Carolina	Recruiting
Duke University Medical Center (Duke South Clinics) -40 Duke Medicine Cir	Durham	North Carolina	Recruiting
Hightower Clinical - Oklahoma Cancer Center	Oklahoma City	Oklahoma	Recruiting
Cancer Care Associates of York	York	Pennsylvania	Not Yet Recruiting
Intermountain Medical Center	Murray	Utah	Not Yet Recruiting
Velocity Clinical Research - Seattle	Seattle	Washington	Recruiting
Washington State Univ Elson S. Floyd College of Medicine	Spokane	Washington	Recruiting
Gallipoli Medical Research	Chandler	Queensland	Recruiting
Royal Brisbane and Women's Hospital	Brisbane	Australia	Recruiting
Monash Medical Centre	Clayton	Australia	Not Yet Recruiting
Trials West	Perth	Australia	Recruiting
Westmead Hospital for Medical Research	Westmead	Australia	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
Ghent University Hospital	Ghent	West Vlaanderen	Recruiting
Universitair Ziekenhuis Antwerpen (UZA)	Edegem	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

+ 60 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07332091 on ClinicalTrials.gov ↗ ← All trials in Ireland