A Phase 2 interventional study of Vamifeport and Placebo in Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis, sponsored by CSL Behring. Active, not recruiting at 100 sites in 18 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by CSL Behring (part of CSL Limited) · 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.
55 studies on the registry are indexed under Hemochromatosis; 4 are open to participants now.
This study's planned enrollment of 84 is above the median of 61 across 30 interventional studies indexed under Hemochromatosis.
Browse Hemochromatosis studies →CSL Behring is the lead sponsor of 145 studies on the registry; 17 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.
First submitted after CSL Behring became part of CSL Limited.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Evidence of iron overload as shown by:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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, Statistical analysis plan
Time frame: Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
Access criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: VIT-2763