EUCTR2018-004689-32-SE进行中（未招募）1 期
A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 45
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
- All
入选标准
- •1. The subject must voluntarily sign an Institutional Review Board (IRB)/Independent Ethics Committee/Research Ethics Board approved informed consent form after all relevant aspects of the study have been explained and discussed with the subject.
- •2. The subject has Fabry disease as confirmed at screening by the following criteria using a dried blood spot (DBS) assay:
- •? For male subjects, Fabry disease is confirmed by a deficiency of GLA activity and a mutation in the GLA gene.
- •? For female subjects, Fabry disease is confirmed by a mutation in the GLA gene.
- •3. The subject is 18 to 65 years of age, inclusive.
- •4. Female subjects must have a negative pregnancy test at screening.
- •5. Female subjects of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and for at least 14 days after the final study infusion; the methods of acceptable contraception are listed in the protocol.
- •6. The subject is deemed, as determined by the investigator, to have adequate general health to undergo the specified protocol-related procedures and to have no safety or medical contraindications for participation.
- •7. The subject has not received any treatment (approved or investigational) specific to Fabry disease, such as ERT, chaperone therapy, or substrate reduction therapy.
- •8. The subject must have an eGFR of 45 to 120 mL/min/1.73 m2; eGFR will be calculated by a Shire-designated laboratory using the CKD-EPI formula. If the eGFR measurement at screening is not within the stipulated range, a second eGFR measurement may be completed and, if in range, used as the screening value. If a second measurement is taken, a minimum of 1 week and maximum of 30 days should separate it from the first. This inclusion criterion follows the European Guidelines for Treatment of Fabry Disease (Biegstraaten et al., 2015) and Kidney Disease Improving Global Outcomes guidelines for classification of renal disease (Kidney Disease Improving Global Outcomes (KDIGO), 2013).
- •9. The subject has left ventricular hypertrophy (LVH), where LVH is defined as left ventricular mass index (LVMI) >50 g/m2.7 confirmed by cardiac magnetic resonance imaging (cMRI) at screening. The cMRI value at screening will serve as the baseline value.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 42
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 3
排除标准
- •1. In the opinion of the investigator, the subject’s life expectancy is =5 years.
- •2. The subject has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis, or has any signs or symptoms of end stage renal disease.
- •3. Protein/creatinine ratio (PCR) >0.15 mg/mg female and >0.14 mg/mg male.
- •4. Subjects who have clinically relevant history of allergy or signs or symptoms of severe hypersensitivity (including hypersensitivity to the REPLAGAL active substance or any of the excipients), which in the investigator’s judgment, will substantially increase the subject’s risk if he or she participates in the study.
- •5. Cardiac fibrosis involving more than 2 segments, as determined by cMRI at screening.
- •6. In the opinion of the investigator, the subject has non-Fabry disease-related cause of end-organ (renal, cardiac, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by cardiac and/or renal measures.
- •7. The subject has a positive test at screening for hepatitis B surface antigen (HBsAg), positive test for hepatitis B core antibody (HBcAb), positive test for hepatitis C (HCV) antibody with confirmation by HCV-ribonucleic acid polymerase chain reaction testing, or positive test for human immunodeficiency virus (HIV) antibody.
- •8. Treatment with REPLAGAL at any time prior to the study with REPLAGAL.
- •9. Prior treatment with any of the following medications:
- •? FABRAZYME (agalsidase beta) and its biosimilars
- •? GLYSET® (miglitol)
- •? ZAVESCA® (miglustat)
- •? CERDELGA® (eliglustat)
- •? GALAFOLD® (migalastat)
- •? Any investigational product for treatment of Fabry disease.
- •? Chloroquine
- •? Amiodarone
- •? Monobenzone
- •? Gentamicin.
- •10. The subject is pregnant or lactating.
- •11. The subject has a body mass index >39 kg/m2. (BMI = kg/m2).
- •12. The subject is treated or has been treated with any investigational drug within 30 days prior to study start.
- •13. The subject is unable to understand the nature, scope, and possible consequences of the study.
- •14. The subject is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or isotherwise unlikely to complete
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