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

Conditions

• Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Vamifeport — DRUG
  Vamifeport capsule administered orally.
• Placebo — DRUG
  Placebo capsule matching IP administered orally.

Study Details

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.

Key Dates

Start date

Jan 22, 2026

Status verified

May 2026

Primary completion

Mar 6, 2028

Completion

Apr 6, 2028

Study Design

Enrollment

84 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Vamifeport Low Dose
  Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
• Experimental: Vamifeport High Dose
  Participants will receive a high dose of vamifeport orally, BID up to Day 360.
• Placebo Comparator: Placebo
  Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.

Primary Outcome Measure

Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC) [ Time Frame: At Baseline and Day 360 ]

Central Contacts

• Trial Registration Coordinator
  +16108784697
  [email protected]

Locations (18)

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