Recruiting
Phase 2

Vamifeport

Sponsor:

CSL Behring

Code:

NCT07332091

Conditions

Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Vamifeport

Placebo

Study Details

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.

Conditions

Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

Study ID

NCT07332091

Start date

Jan 22, 2026

Status verified date

Aug, 2026

Completion date

Apr 6, 2028

Anticipated

Primary completion date

Mar 6, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

84 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Vamifeport Low Dose

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

experimental: Vamifeport High Dose

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

placebo comparator: Placebo

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

Interventions

Vamifeport

Vamifeport capsule administered orally.

Placebo

Placebo capsule matching IP administered orally.

Primary outcome measure

  • Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC) [ Time Frame: At Baseline and Day 360 ]

Central Contacts and Locations

Central contacts

Trial Registration Coordinator

+16108784697clinicaltrials@cslbehring.com

Locations

Green Leaf Clinical Trials

Recruiting

Jacksonville, Florida, United States, 32258

Indiana University Health University Hospital

Recruiting

Indianapolis, Indiana, United States, 46202-5149

Ochsner Medical Complex - High Grove

Recruiting

Baton Rouge, Louisiana, United States, 70836

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

Recruiting

Bethesda, Maryland, United States, 20817

James M. Stockman Cancer Institute

Recruiting

Frederick, Maryland, United States, 21702-4337

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

Recruiting

Duluth, Minnesota, United States, 55805

University of North Carolina at Chapel Hill

Recruiting

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

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

Recruiting

Durham, North Carolina, United States, 27710-4000

Hightower Clinical - Oklahoma Cancer Center

Recruiting

Oklahoma City, Oklahoma, United States, 73174

Velocity Clinical Research - Seattle

Recruiting

Seattle, Washington, United States, 98105

Washington State Univ Elson S. Floyd College of Medicine

Recruiting

Spokane, Washington, United States, 99202-2131

More Information

Sponsor

CSL Behring

Last update posted

Aug 20, 2026

Last verified

Aug, 2026

Keywords

  • Small Molecule Ferroportin Inhibitor
  • Hereditary hemochromatosis
  • Iron overload

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by CSL Behring on 2026-08-20.