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NCT Number: NCT07332091

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

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.

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Key information

Conditions

Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hemochromatosis Iron Metabolism Disorders Iron Overload Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Royal Brisbane and Women's Hospital, Brisbane, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Vamifeport

Drug

Vamifeport capsule administered orally.

Placebo

Drug

Placebo capsule matching IP administered orally.

Primary outcomes

  1. Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)

    Time frame: At Baseline and Day 360

Secondary outcomes

  1. Number of participants with treatment-emergent adverse events (TEAEs)

    Time frame: Up to Day 390

  2. Percentage of participants with TEAEs

    Time frame: Up to Day 390

  3. Number of participants with treatment-emergent serious adverse events (SAEs)

    Time frame: Up to Day 390

  4. Percentage of participants with treatment-emergent SAEs

    Time frame: Up to Day 390

  5. 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

    Clinical safety laboratory tests will include hematology, biochemistry and coagulation.

  6. 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

    Clinical safety laboratory tests will include hematology, biochemistry and coagulation.

  7. Change from baseline in transferrin saturation (TSAT) (measured at trough)

    Time frame: From Baseline to Day 360

  8. Number of participants with TSAT less than or equal to (<=) 45% (measured at trough)

    Time frame: From Baseline to Day 360

  9. Percentage of participants with TSAT <= 45% (measured at trough)

    Time frame: From Baseline to Day 360

  10. Number of participants with 25% reduction in MRI-based LIC

    Time frame: At Day 180 and 360

  11. Number of participants with 50% reduction in MRI-based LIC

    Time frame: At Day 180 and Day 360

  12. Number of participants with TSAT ≤ 45% or MRI-based LIC < 5 milligrams per gram (mg/g)

    Time frame: At Day 360

  13. Number of participants with TSAT ≤ 45% or MRI-based LIC < 2 mg/g

    Time frame: At Day 360

  14. Change from baseline in joint pain score in a visual analog scale (VAS)

    Time frame: From Baseline to Day 360

    The VAS is a single-item questionnaire. Participants will be asked to record their joint pain at its worst over the previous week, from 0 (No pain) to 10 (Worst possible pain).

  15. Change from baseline in modified fatigue impact scale (MFIS) total score

    Time frame: From Baseline to Day 360

    Participants will be asked to read a list of 21 statements and assess how often fatigue has affected them in terms of physical, cognitive, and psychosocial functioning, over the previous 4 weeks, using a 5-point scale from 0 (Never) to 4 (Almost always). The total score is obtained by summing the scores of all the 21 items. Higher scores indicate a greater impact of fatigue on a participant's activities.

  16. Change from baseline in health-related quality of life: EuroQoL 5-dimension 5-level instrument (EQ-5D-5L)

    Time frame: From Baseline to Days 180, 360, and 390

    The EQ-5D-5L is a standardized measure of health status that provides a simple, generic measure of health for clinical and economic appraisal. Participants will complete the questionnaire based on their health on that day. The EQ-5D-5L descriptive profile includes 5 dimensions: mobility, self-care, usual activities, pain / discomfort, and anxiety / depression. Participants will rate each dimension based on 5 levels of severity: no problems, slight problems, moderate problems, severe problems, and extreme problems. A higher score indicates a more severe outcome than a lower score.

  17. Change from baseline in health-related quality of life: VAS (Arthralgia)

    Time frame: From Baseline to Day 180, 360, and 390

    The VAS is a single-item questionnaire. Participants will be asked to record their joint pain at its worst over the previous week, from 0 (No pain) to 10 (Worst possible pain).

  18. Change from baseline in health-related quality of life: Patient global impression of change in clinical status

    Time frame: From Baseline to Day 180, 360, and 390

    The PGI - Change (PGI-C) instrument is a self-reported measure that reflects belief about the efficacy of treatment. Participants will be asked to rate the change in their overall symptoms since they started taking investigational product (IP) on a 5-point scale, ranging from 1 (Much improved) to 5 (Much worse). A lower score reflects an improvement in clinical status.

  19. Change from baseline in health-related quality of life: Patient global impression of severity

    Time frame: From Baseline to Day 180, 360, and 390

    The PGI - Severity (PGI-S) instrument is a self-reported questionnaire and consists of 1 item that measures disease severity. Participants will be asked to rate the severity of their overall symptoms over the previous week on a 5-point scale from 1 (None) to 5 (Very severe). A higher score reflects a more severe outcome than a lower score.

  20. Change from baseline in health-related quality of life: MFIS physical, cognitive, and psychosocial subscales

    Time frame: From Baseline to Day 180, 360, and 390

    Participants will be asked to read a list of 21 statements and assess how often fatigue has affected them in terms of physical, cognitive, and psychosocial functioning, over the previous 4 weeks, using a 5-point scale from 0 (Never) to 4 (Almost always).

    Items on the MFIS will be aggregated into 3 subscales (Physical, Cognitive, or Psychosocial). Higher scores indicate a greater impact of fatigue on a participant's activities.

  21. Vamifeport plasma concentrations after a single dose

    Time frame: At Day 1

  22. Vamifeport plasma concentrations at steady state

    Time frame: At Days 15, 180, and 360

  23. Change from baseline in total serum iron (measured at trough)

    Time frame: From Baseline to Day 360

  24. Change from baseline in serum ferritin (measured at trough)

    Time frame: From Baseline to Day 360

  25. Frequency of rescue therapy use

    Time frame: Up to Day 390

    For assessment of this outcome measure, data will be collected retrospectively from 1 year before baseline as well as during the study (up to Day 390).

  26. Duration of rescue therapy use

    Time frame: Up to Day 390

    For assessment of this outcome measure, data will be collected retrospectively from 1 year before baseline as well as during the study (up to Day 390).

  27. Time to first use of rescue therapy

    Time frame: Up to Day 360

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Registration Coordinator

CONTACT

[email protected]

+16108784697

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study 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)

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Jan 12, 2026
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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