Recruiting NCT07332091
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Vamifeport, Placebo.
- Who it may be relevant to
- Registry conditions: Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Austria, Belgium, Canada +13
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study of the Efficacy and Safety of Vamifeport in Adult Subjects With HFE-related Hereditary Hemochromatosis (FERROCLEAR Study)
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.
Interventions
- Drug Vamifeport
Vamifeport capsule administered orally. - Drug Placebo
Placebo capsule matching IP administered orally.
Primary outcome measures
- Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC) [Time frame: At Baseline and Day 360]
Secondary outcome measures (12)
- Number of participants with treatment-emergent adverse events (TEAEs) [Time frame: Up to Day 390]
- Percentage of participants with TEAEs [Time frame: Up to Day 390]
- Number of participants with treatment-emergent serious adverse events (SAEs) [Time frame: Up to Day 390]
- Percentage of participants with treatment-emergent SAEs [Time frame: Up to Day 390]
- Number of participants with clinically significant change from baseline in clinical safety laboratory tests and 12-lead electrocardiogram (ECG) [Time frame: From Baseline to Day 390]
- Percentage of participants with clinically significant change from baseline in clinical safety laboratory tests and 12-lead ECG [Time frame: From Baseline to Day 390]
- Change from baseline in transferrin saturation (TSAT) (measured at trough) [Time frame: From Baseline to Day 360]
- Number of participants with TSAT less than or equal to (<=) 45% (measured at trough) [Time frame: From Baseline to Day 360]
- Percentage of participants with TSAT <= 45% (measured at trough) [Time frame: From Baseline to Day 360]
- Number of participants with 25% reduction in MRI-based LIC [Time frame: At Day 180 and 360]
- Number of participants with 50% reduction in MRI-based LIC [Time frame: At Day 180 and Day 360]
- Number of participants with TSAT ≤ 45% or MRI-based LIC < 5 milligrams per gram (mg/g) [Time frame: At Day 360]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 19 centers
- Banner MD Anderson — Gilbert
- Infinity Clinical Trials — San Diego
- Medical Oncology Associates of San Diego — San Diego
- Green Leaf Clinical Trials — Jacksonville
- Indiana University Health University Hospital — Indianapolis
- Ochsner Medical Complex - High Grove — Baton Rouge
- Johns Hopkins University School of Medicine — Baltimore
- American Oncology Partners, PA dba The Center for Cancer and Blood Disorders — Bethesda
- … and 11 more centers
United Kingdom · 12 centers
Center list to be confirmed — check the primary protocol.
France · 11 centers
- Aphp Avicenne — Bobigny
- Chu Dupuytren — Limoges
- CRMR Maladies du Globule Rouge, Hôpital de la Timone — Marseille
- CHU de Montpellier- Hôpital Saint Eloi — Montpellier
- GHRMSA — Mulhouse
- CHU de Bordeaux - Hôpital Haut Leveque — Pessac
- Centre Hospitalier Lyon Sud/Hospices Civils de Lyon — Pierre-Bénite
- Chu Rennes — Rennes
- … and 3 more centers
Spain · 7 centers
- Hospital Universitari Germans Trias i Pujol — Badalona
- Hospital Clinic Barcelona — Barcelona
- HUGC Doctor Negrin — LAS Palmas de GC
- … and 4 more centers
Italy · 6 centers
- ASL Brindisi - Presidio Ospedaliero Di Summa - Perrino — Brindisi
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico — Milan
- University Hospital of Modena — Modena
- Fondazione IRCCS San Gerardo dei Tintori — Monza
- AORN Cardarelli — Naples
- University of Verona - Azienda Ospedaliera Universitaria Integrata Verona — Verona
Australia · 5 centers
- Royal Brisbane and Women's Hospital — Brisbane
- Gallipoli Medical Research — Chandler
- Monash Medical Centre — Clayton
- Trials West — Perth
- Westmead Hospital for Medical Research — Westmead
Belgium · 5 centers
- Universitair Ziekenhuis Antwerpen (UZA) — Edegem
- Ghent University Hospital — Ghent
- UZ Brussel — Jette
- Centre Hospitalier Universitaire de Liège (CHU de Liège) — Liège
- CHU UCL Namur - Site Godinne — Yvoir
Romania · 5 centers
- Spitalul Clinic de Urgenta Prof Dr Agrippa Ionescu-Balotesti — Baloteşti
- Bistrita County Emergency Clinical Hospital — Bistriţa
- Coltea Clinical Hospital — Bucharest
- L'Institute Oncologique Prof. Dr. Ion Chiricuta (IOCN) — Cluj-Napoca
- Prof. Dr.Octavian Fodor Regional Institute of Gastroenterology-Hepatology — Cluj-Napoca
Germany · 4 centers
- Universitätsklinikum Freiburg — Freiburg im Breisgau
- University Hospital Heidelberg — Heidelberg
- EUGASTRO GmbH — Leipzig
- MVZ für Innere Medizin Weinheim — Weinheim
Austria · 3 centers
- Medical University of Innsbruck — Innsbruck
- Ordensklinikum Linz - Barmherzige Schwestern — Linz
- Medical University Vienna — Vienna
Canada · 3 centers
- Libin Cardiovascular Institute University of Calgary — Calgary
- McMaster University-St. Josephs Healthcare Hamilton — Hamilton
- University of Manitoba — Winnipeg
Czechia · 3 centers
- Fakultní Nemocnice Brno — Brno
- Fakultni nemocnice Ostrava — Ostrava
- Institut Klinicke a Experimentalni Mediciny — Prague
Denmark · 3 centers
- Aarhus University Hospital — Aarhus
- Bispebjerg Hospital — Copenhagen
- Copenhagen University Hospital - Hvidovre — Hvidovre
Ireland · 3 centers
- Cork University Hospital — Cork
- Beaumont Hospital — Dublin
- Connolly Hospital Blanchardstown — Dublin
Poland · 3 centers
- Instytut Hematologii i Transfuzjologii — Warsaw
- Specjalistyczny Szpital im. Alfreda Sokolowskiego — Wałbrzych
- Wojewodzki Szpital im. J Gromkowskiego we Wroclawiu — Wroclaw
Switzerland · 3 centers
Center list to be confirmed — check the primary protocol.
Netherlands · 2 centers
- Maastricht UMC — Maastricht
- Radboud UMC — Nijmegen
New Zealand · 2 centers
- Auckland City Hospital — Auckland
- Aotearoa Clinical Trials Trust- Middlemore Hospital — Auckland
Identifiers
NCT: NCT07332091 · CSL624_2001 · 2025-523793-16-00