NCT07332091
Recruiting
Phase 2
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)
Overview
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- CSL Behring
- Enrollment
- 84
- Locations
- 96
- Primary Endpoint
- Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)
Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
Vamifeport Low Dose
Experimental
Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
Intervention: Vamifeport (Drug)
Vamifeport High Dose
Experimental
Participants will receive a high dose of vamifeport orally, BID up to Day 360.
Intervention: Vamifeport (Drug)
Placebo
Placebo Comparator
Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)
Time Frame: At Baseline and Day 360
Secondary Outcomes
- Number of participants with treatment-emergent adverse events (TEAEs)(Up to Day 390)
- Percentage of participants with TEAEs(Up to Day 390)
- Number of participants with treatment-emergent serious adverse events (SAEs)(Up to Day 390)
- Percentage of participants with treatment-emergent SAEs(Up to Day 390)
- Number of participants with clinically significant change from baseline in clinical safety laboratory tests and 12-lead electrocardiogram (ECG)(From Baseline to Day 390)
- Percentage of participants with clinically significant change from baseline in clinical safety laboratory tests and 12-lead ECG(From Baseline to Day 390)
- Change from baseline in transferrin saturation (TSAT) (measured at trough)(From Baseline to Day 360)
- Number of participants with TSAT less than or equal to (<=) 45% (measured at trough)(From Baseline to Day 360)
- Number of participants with TSAT ≤ 45% or MRI-based LIC < 5 milligrams per gram (mg/g)(At Day 360)
- Number of participants with TSAT ≤ 45% or MRI-based LIC < 2 mg/g(At Day 360)
- Change from baseline in health-related quality of life: EuroQoL 5-dimension 5-level instrument (EQ-5D-5L)(From Baseline to Days 180, 360, and 390)
- Percentage of participants with TSAT <= 45% (measured at trough)(From Baseline to Day 360)
- Number of participants with 25% reduction in MRI-based LIC(At Day 180 and 360)
- Number of participants with 50% reduction in MRI-based LIC(At Day 180 and Day 360)
- Change from baseline in health-related quality of life: VAS (Arthralgia)(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(From Baseline to Day 180, 360, and 390)
- Change from baseline in health-related quality of life: MFIS physical, cognitive, and psychosocial subscales(From Baseline to Day 180, 360, and 390)
- Vamifeport plasma concentrations after a single dose(At Day 1)
- Change from baseline in total serum iron (measured at trough)(From Baseline to Day 360)
- Change from baseline in joint pain score in a visual analog scale (VAS)(From Baseline to Day 360)
- Change from baseline in modified fatigue impact scale (MFIS) total score(From Baseline to Day 360)
- Change from baseline in health-related quality of life: Patient global impression of severity(From Baseline to Day 180, 360, and 390)
- Vamifeport plasma concentrations at steady state(At Days 15, 180, and 360)
- Change from baseline in serum ferritin (measured at trough)(From Baseline to Day 360)
- Frequency of rescue therapy use(Up to Day 390)
- Duration of rescue therapy use(Up to Day 390)
- Time to first use of rescue therapy(Up to Day 360)
Investigators
Study Sites (96)
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