A Phase 2 interventional study of Vamifeport and Placebo in Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis, sponsored by CSL Behring. Recruiting at 100 sites in 18 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-16.
Sponsored by CSL Behring · Phase 2, Interventional, and Treatment
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.
Evidence of iron overload as shown by:
Exclusion Criteria:
Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
Drug: Vamifeport
Participants will receive a high dose of vamifeport orally, BID up to Day 360.
Drug: Vamifeport
Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.
Drug: Placebo
Vamifeport capsule administered orally.
Placebo capsule matching IP administered orally.
Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)
Time frame: At Baseline and Day 360
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)
Clinical safety laboratory tests will include hematology, biochemistry and coagulation.
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
Clinical safety laboratory tests will include hematology, biochemistry and coagulation.
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
Number of participants with TSAT ≤ 45% or MRI-based LIC < 2 mg/g
Time frame: At Day 360
Change from baseline in joint pain score in a visual analog scale (VAS)
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).
Time frame: From Baseline to Day 360
Change from baseline in modified fatigue impact scale (MFIS) total score
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.
Time frame: From Baseline to Day 360
Change from baseline in health-related quality of life: EuroQoL 5-dimension 5-level instrument (EQ-5D-5L)
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.
Time frame: From Baseline to Days 180, 360, and 390
Change from baseline in health-related quality of life: VAS (Arthralgia)
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).
Time frame: From Baseline to Day 180, 360, and 390
Change from baseline in health-related quality of life: Patient global impression of change in clinical status
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.
Time frame: From Baseline to Day 180, 360, and 390
Change from baseline in health-related quality of life: Patient global impression of severity
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.
Time frame: From Baseline to Day 180, 360, and 390
Change from baseline in health-related quality of life: MFIS physical, cognitive, and psychosocial subscales
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.
Time frame: From Baseline to Day 180, 360, and 390
Vamifeport plasma concentrations after a single dose
Time frame: At Day 1
Vamifeport plasma concentrations at steady state
Time frame: At Days 15, 180, and 360
Change from baseline in total serum iron (measured at trough)
Time frame: From Baseline to Day 360
Change from baseline in serum ferritin (measured at trough)
Time frame: From Baseline to Day 360
Frequency of rescue therapy use
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).
Time frame: Up to Day 390
Duration of rescue therapy use
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).
Time frame: Up to Day 390
Time to first use of rescue therapy
Time frame: Up to Day 360
Plan to share: Yes — CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Supporting information: Study protocol, Sap
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