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

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

  • Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

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