Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis

CSL Behring

Status and phase: Enrolling; Phase 2

Conditions: Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

Treatments: Drug: Placebo; Drug: Vamifeport

Study type: Interventional

Funder types: Industry

Identifiers

NCT07332091

CSL624_2001

2025-523793-16-00 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 84 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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84 participants in 3 patient groups, including a placebo group

Vamifeport Low Dose

Experimental group

Description:

Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.

Treatment:

Drug: Vamifeport

Vamifeport High Dose

Experimental group

Description:

Participants will receive a high dose of vamifeport orally, BID up to Day 360.

Treatment:

Drug: Vamifeport

Placebo

Placebo Comparator group

Description:

Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.

Treatment:

Drug: Placebo