NCT07332091招募中2 期
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)
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- CSL Behring
- 入组人数
- 84
- 试验地点
- 112
- 主要终点
- Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 2.4 and 16 mg/g (43.0 and 286.5 mmol/kg) dry weight (dw) at Screening.
- •Body mass index between 18.5 and 34.9 kilograms per meter squared (kg/m^2).
排除标准
- •Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive placebo matching vamifeport low and high doses orally, BID up to Day 360.
干预措施: Placebo (Drug)
Vamifeport Low Dose
Experimental
Participants will receive a low dose of vamifeport orally, twice daily (BID) up to Day 360.
干预措施: Vamifeport (Drug)
Vamifeport High Dose
Experimental
Participants will receive a high dose of vamifeport orally, BID up to Day 360.
干预措施: Vamifeport (Drug)
结局指标
主要结局
Change from baseline in magnetic resonance imaging (MRI)-based liver iron concentration (LIC)
时间窗: At Baseline and Day 360
次要结局
- Number of participants with TSAT less than or equal to (<=) 45% (measured at trough)(From Baseline to Day 360)
- Percentage of participants with TSAT <= 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)
- 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)
- 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)
- Number of participants with TSAT ≤ 45% or MRI-based LIC < 2 mg/g(At Day 360)
- 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)
研究者
研究点 (112)
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