EUCTR2019-000065-20-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 effects of switching to agalsidase beta (Fabrazyme®) versus continuing on migalastat (Galafold®) in male patients with classic Fabry disease - BCLEAR2
Sanofi Aventis Groupe (SAG)0 个研究点目标入组 35 人开始时间: 2019年10月31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 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 migalastat for a minimum of 6 months up to a maximum of 12 months at baseline.
- •Participants are naive to agalsidase beta.
- •Participants with 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 average 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 during screening period.
- •Participant’s medical records (including eGFR values) available and accessible during the study period.
- •Male, only male participants will be included in the study.
- •Participants and/or participant’s legal representative capable of giving signed informed consent as described in Appendix 1 (Section 10.1.3) which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. For potential participants aged 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 a history of renal transplantation, or who are currently receiving hemo- or peritoneal dialysis; participants with comorbid nonFabry nephropathies (e.g., diabetic nephropathy, glomerulonephritis, etc.).
- •Participants with rapid renal decline: Loss of >6 mL/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 diagnosed with diabetes.
- •Participants with history of anaphylaxis to 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 3 years with agalsidase alfa prior to initiating migalastat therapy.
- •Exposure to any investigational study intervention other than migalastat in the last 4 weeks or 5 half-lives, whichever is longer, prior to screening visit or concomitant enrollment in any other clinical study involving an investigational study intervention (patients participating in any migalastat clinical study may be considered upon discontinuation and meeting eligibility criteria).
- •Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants 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 who 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.
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