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Purpose

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.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 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).

Exclusion Criteria

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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
This is multicenter, randomized, placebo-controlled double-blind, parallel-group study.
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Vamifeport Low Dose 		Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
  • Drug: Vamifeport
    Vamifeport capsule administered orally.
Experimental
Vamifeport High Dose 		Participants will receive a high dose of vamifeport orally, BID up to Day 360.
  • Drug: Vamifeport
    Vamifeport capsule administered orally.
Placebo Comparator
Placebo 		Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.
  • Drug: Placebo
    Placebo capsule matching IP administered orally.

Recruiting Locations

Green Leaf Clinical Trials
Jacksonville, Florida 32258

Indiana University Health University Hospital
Indianapolis, Indiana 46202-5149

American Oncology Partners, PA dba The Center for Cancer and Blood Disorders
Bethesda, Maryland 20817

Aspirus St. Luke's Clinic - Duluth - Oncology & Hematology
Duluth, Minnesota 55805

Hightower Clinical - Oklahoma Cancer Center
Oklahoma City, Oklahoma 73174

Washington State Univ Elson S. Floyd College of Medicine
Spokane, Washington 99202-2131

More Details

Status
Recruiting
Sponsor
CSL Behring

Study Contact

Trial Registration Coordinator
+16108784697
clinicaltrials@cslbehring.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.