Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
Phase 2 – studying effectiveness and dosage
Conditions:
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Sponsor: CSL Behring
trial.available_in:
БГ
Overview
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.
Who can participate
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.
Locations
16
Australia (1)
Royal Brisbane and Women's Hospital
Brisbane
NOT_YET_RECRUITING
Belgium (1)
Universitair Ziekenhuis Antwerpen (UZA)
Edegem
NOT_YET_RECRUITING
Canada (1)
Libin Cardiovascular Institute University of Calgary
Calgary
NOT_YET_RECRUITING
Czech Republic (1)
Fakultní Nemocnice Brno
Brno
NOT_YET_RECRUITING
Denmark (1)
Aarhus University Hospital
Aarhus
NOT_YET_RECRUITING
France (1)
Aphp Avicenne
Bobigny
NOT_YET_RECRUITING
Ireland (1)
Cork University Hospital
Cork
NOT_YET_RECRUITING
Italy (1)
ASL Brindisi - Presidio Ospedaliero Di Summa - Perrino
Brindisi
NOT_YET_RECRUITING
Netherlands (1)
Maastricht UMC
Maastricht
NOT_YET_RECRUITING
New Zealand (1)
Auckland City Hospital
Auckland
NOT_YET_RECRUITING
Poland (1)
Instytut Hematologii i Transfuzjologii
Warsaw
NOT_YET_RECRUITING
Romania (1)
Spitalul Clinic de Urgenta Prof Dr Agrippa Ionescu-Balotesti