Health TrialsbyHealthPanel

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

ConditionHomeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorCSL Behring

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adult (≥ 18 years) and has provided written informed consent.

Confirmed diagnosis of HFE-HH in medical history.

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

Disqualifiers

Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Vamifeport

    Drug

    Vamifeport capsule administered orally.

  • Placebo

    Drug

    Placebo capsule matching IP administered orally.

Treatment groups

84 Participants
are divided into 3 treatment groups
Group A: Vamifeport Low DoseExperimental treatment 1 intervention
Group B: Vamifeport High DoseExperimental treatment 1 intervention
Group C: PlaceboPlacebo comparator 1 intervention

Trial outcomes

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

CSL Behring

Lead sponsor

Source ClinicalTrials.gov
Apply now