Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorCSL Behring
About this trial
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 criteria
Qualifiers
Adult (≥ 18 years) and has provided written informed consent.
Confirmed diagnosis of HFE-HH in medical history.
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
Disqualifiers
Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.
Trial design
Treatments tested in this trial
- Vamifeport
- Placebo
Treatment groups
84 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov