EUCTR2019-000064-21-NO进行中（未招募）1 期
A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical benefits of agalsidase beta (Fabrazyme®) in male patients with classic Fabry disease switching from agalsidase alfa (Replagal®)
Sanofi Aventis Groupe (SAG)0 个研究点目标入组 35 人开始时间: 2019年8月15日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 35
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •Male participant must be 16 to 45 years of age inclusive, at the time of signing the informed consent.
- •Participants who are diagnosed with classic Fabry disease based on phenotype, presence or absence of characteristic Fabry disease symptoms including neuropathic pain, clustered angiokeratoma and/or cornea verticillata, leucocyte a-GAL A enzyme activity (3% or less compared to control), and genotype (optional).
- •Participants who are currently receiving agalsidase alfa for a minimum of 6 months at an average dose of 0.2 mg/kg every other week (ie, every 2 weeks) at baseline.
- •Participants who are naïve to agalsidase beta.
- •Participants with estimated glomerular filtration rate (eGFR) =60 mL/min/1.73 m^2 at screening and baseline.
- •Proteinuria level as measured by 2 separate, morning, clean-catch urine samples taken a few days apart demonstrating an averaged urine protein-creatinine ratio of <0.5 (ie, <500 mg protein per 1 g creatinine) between the 2 samples. For participants on angiotensin-converting enzyme inhibitors (ACEIs) or angiotensin receptor blockers (ARBs), the criterion is to be met both prior and after a temporary interruption of ACEIs/ARBs for 4 weeks.
- •Participants with plasma lyso-GL3 levels >20 ng/mL on 2 consecutive samples taken at least 4 weeks apart.
- •Participant’s medical records (including eGFR values) available and accessible during the study period.
- •Participant and/or participant’s legal representative has given signed informed consent as described in the protocol which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. For potential participants age 16 to 18 years, a parent or legal representative is required to sign the ICF, and the potential participant is also required to sign an informed assent form.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 4
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 31
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Participants with severe renal impairment (end-stage renal disease, dialysis, or renal transplantation) and/or nephropathies (including diabetic).
- •Participants with rapid renal decline: Loss of >6mL/min/1.73 m^2 at screening compared to the most recent eGFR value approximately 12 months prior to screening.
- •Participants with advanced cardiac failure (Stage D).
- •Participants with bleeding disorder, prior history of unexplained bleeding episodes, or receiving mandatory anticoagulants or antiplatelets for any indication not allowing interruption of therapy for renal biopsy.
- •Participants with diagnosed diabetes.
- •Participants with history of anaphylaxis to Enzyme Replacement Therapy (ERT).
- •Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
- •Participants treated for more than 5 years with agalsidase alfa at an average dose of 0.2 mg/kg every other week (ie, every 2 weeks) prior to randomization.
- •Exposure to migalastat or any investigational study intervention, except agalsidase alfa, for Fabry disease in the last 5 years prior to study participation. Patients who previously participated in any agalsidase alfa clinical study will be eligible if they meet other criteria.
- •Exposure to any investigational drugs in the last 4 weeks or 5 halflives, whichever is longer, prior to screening visit or concomitant enrollment in any other clinical study involving an investigational study treatment.
- •Individuals accommodated in an institution because of regulatory or legal order; prisoners or subjects who are legally institutionalized.
- •Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
- •Participants are dependent on the Sponsor or Investigator or deemed vulnerable for any reason (in conjunction with Section 1.61 of the International Council for Harmonisation Good Clinical Practice [ICH-GCP] Ordinance E6).
- •Participants who are employees of the clinical study center or other individuals directly involved in the conduct of the study, or immediate family members of such individuals.
- •Any specific situation during study implementation/course that may raise ethics consideration
研究者
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