2026-525207-27-00招募中3 期
A multicenter, open-label, single-arm, baseline-controlled trial to assess the efficacy and safety of lucerastat in treatment-naïve/pseudo-naïve adult male participants with Fabry disease
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 8
- 试验地点
- 4
- 主要终点
- Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).
研究概览
简要总结
To evaluate the effect of lucerastat on kidney Globotriaosylceramide (Gb3) burden in treatment naïve/pseudo-naïve adult male participants with Fabry Disease using a quantitative scoring system
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Fabry Disease: – Plasma and/or leukocyte α-galactosidase A (α-GalA) < 1% mean normal levels or – Known “pathogenic” or “likely pathogenic” Gene coding for α-galactosidase A (GLA) variant with a low level (i.e., < 30% mean normal levels) of plasma and/or leukocyte α-GalA.
- •History of at least one of the following clinical manifestations of Fabry Disease: – Neuropathic pain – Cornea verticillata – Angiokeratoma
- •Treatment-naïve or pseudo-naïve i.e. without prior treatment with an approved or any investigational therapy for Fabry Disease within at least 6 months prior to screening.
- •Plasma globotriaosylsphingosine ≥ 20 ng/ml (as assessed centrally).
- •Screening estimated glomerular filtration rate (eGFR) (central laboratory) ≥ 45 mL/min/1.73 m2.
排除标准
- •Any intercurrent condition or concomitant therapy considered a contraindication for kidney biopsy, as per local standard of care, or in the investigator’s opinion may preclude accurate interpretation of trial data.
- •Any other known factor or disease that might interfere with treatment compliance, trial conduct, or interpretation of the results, such as drug or alcohol dependence or psychiatric disease including any of the following: – moderate depression (defined as Beck Depression Inventory-II [BDI-II] score > 20 and ≤ 28) if the participant does not agree to a consultation before Visit 2 with an appropriately qualified mental health professional or – severe depression (i.e., BDI-II score > 28) or – suicidal ideation at screening or history of suicide attempt or behavior within 12 months prior to screening visit.
- •Previous exposure to gene or cell therapy.
- •Use of cationic amphiphilic drugs, such as amiodarone or hydroxychloroquine that may preclude accurate interpretation of kidney biopsy data within 6 months prior to screening.
- •Urine albumin-to-creatinine ratio > 300 mg/g at screening (central laboratory) unless treated with background therapy, such as Angiotensin-converting enzyme inhibitors, Angiotensin receptor blocker or Sodium-glucose cotransporter 2 inhibitors, as per local practice.
- •Inherited or acquired coagulopathy, uncorrected bleeding disorders, international normalized ratio > 1.5, platelet count < 50,000/μL or inability to safely hold anticoagulants or antiplatelet therapy as applicable per local practice (usually 1–2 days for anticoagulants and 3–7 days for antiplatelets).
- •Hemoglobin level < 9.0 g/dL at screening.
- •History of acute kidney injury within 12 months prior to screening visit.
- •Documented poorly controlled diabetes mellitus (i.e., Hemoglobin A1c > 8.0% at screening as reported by the central laboratory).
- •History of cerebrovascular event (e.g. stroke, transient ischemic attack), cardiovascular event (e.g., myocardial infarction, unstable angina), cardiac surgery (e.g., coronary artery bypass graft, valvular repair/replacement) or percutaneous coronary intervention within 6 months prior to screening.
- •Congestive heart failure New York Heart Association class IV or hospitalization for heart failure within 3 months prior to screening.
- •Implementation of cardiac device (e.g., pacemaker, implantable cardioverter defibrillator, cardiac resynchronization therapy device) or hospitalization for arrhythmia within 6 weeks prior to screening.
结局指标
主要结局
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).
次要结局
- Change from baseline to Month 18 in plasma Gb3 concentration.
研究者
Idorsia Clinical Trials Information
Scientific
Idorsia Pharmaceuticals Ltd.
研究点 (4)
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