Condition

HFE-related Hereditary Hemochromatosis

Purpose of the study

To assess the effect of vamifeport treatment on MRI-based LIC in adult subjects with HFE-HH.

Official Title

A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study of Vamifeport in Adult Subjects with HFE-related Hereditary Hemochromatosis.

Who is eligible to participate?

Inclusion Criteria

• Provides written informed consent and is willing and able to adhere to all protocol requirements.
• Confirmed diagnosis of HFE-HH in medical history, ie, homozygosity in the p.C282Y variant in the HFE gene. 3. Subjects with iron overload as shown by:
  • TSAT ≥ 45% (confirmed at 2 visits, at least 14 days apart) at Screening.
  • Serum ferritin ≥ 200 ng/mL but < 5000 ng/mL (confirmed at 2 visits, at least 14 days apart) at Screening.
  • MRI-based LIC between 5 to 16 mg/g dw.
• ≥ 18 years of age.
• Body mass index between 18.5 and 32 kg/m2.
• Investigator believes that the subject understands the nature, scope, and possible consequences of the study.

Exclusion Criteria

• Clinically relevant laboratory abnormalities at Screening.
• Any clinically relevant abnormal 12-lead ECG finding at Screening or Baseline, including but not limited to the following:
• Known contraindications for MRI procedures, such as the presence of metallic implants (including metal aneurysm clips, surgical clips, pins, plates, screws), pacemakers, drug infusion pumps, neurostimulators, cochlear implant, or certain medical devices that are not MRI compatible or safe. Other contraindications include claustrophobia.
• Known liver cirrhosis.
• Presence or history of malignancy (except excised basal cell or squamous cell carcinoma of the skin) within the last 10 years.
• History of hyposplenism / splenectomy.
• History or evidence of clinically significant cardiac or cardiovascular disease.
• Pregnant (eg, positive serum pregnancy test) or childbearing potential subjects unwilling to use a highly effective method of contraception during the study to the EOS Visit.
• Current or recent history of alcohol dependence or drug abuse within 6 months prior to Screening as indicated by medical history or a positive alcohol or drug test.
• Known sensitivity or contraindications to any components of the IP to be administered.
• Participation in any other clinical study with an IP within 30 days prior to Screening.

Register your interest

To register your interest or find out more information about this clinical trial, please email: enquiries@gallipoliresearch.org.au. 

Study location

Gallipoli Medical Research, Greenslopes Private Hospital, Brisbane