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 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
- 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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
Green Leaf Clinical Trials
Jacksonville, Florida 32258
Jacksonville, Florida 32258
Indiana University Health University Hospital
Indianapolis, Indiana 46202-5149
Indianapolis, Indiana 46202-5149
Ochsner Medical Complex - High Grove
Baton Rouge, Louisiana 70836
Baton Rouge, Louisiana 70836
American Oncology Partners, PA dba The Center for Cancer and Blood Disorders
Bethesda, Maryland 20817
Bethesda, Maryland 20817
James M. Stockman Cancer Institute
Frederick, Maryland 21702-4337
Frederick, Maryland 21702-4337
Aspirus St. Luke's Clinic - Duluth - Oncology & Hematology
Duluth, Minnesota 55805
Duluth, Minnesota 55805
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina 27517-7518
Chapel Hill, North Carolina 27517-7518
Duke University Medical Center (Duke South Clinics) -40 Duke Medicine Cir
Durham, North Carolina 27710-4000
Durham, North Carolina 27710-4000
Hightower Clinical - Oklahoma Cancer Center
Oklahoma City, Oklahoma 73174
Oklahoma City, Oklahoma 73174
Velocity Clinical Research - Seattle
Seattle, Washington 98105
Seattle, Washington 98105
Washington State Univ Elson S. Floyd College of Medicine
Spokane, Washington 99202-2131
Spokane, Washington 99202-2131
More Details
- Status
- Recruiting
- Sponsor
- CSL Behring