EUCTR2022-004246-35-Outside-EU/EEA进行中（未招募）1 期
An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese 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）
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Subject and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the subject. For the subjects <18 years old, subjects will give assent AND their parent(s)/legally authorized representative should sign the ICF accordingly.
- •2. The subject has confirmed diagnosis of Fabry disease as determined by the investigator, according to medical record including:
- •For male subject, Fabry disease is confirmed by a deficiency of GLA activity and a mutation in the GLA gene
- •For female subject, Fabry disease is confirmed by a mutation in the GLA gene.
- •3. The subject is 7 to 65 years of age, inclusive, at screening.
- •4. Female subjects of childbearing potential must have a negative pregnancy test at screening.
- •5. Female subjects of childbearing 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 investigational product infusion
- •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 adult subject (=18 years old) must have an eGFR of 45 to 120 mL/min/1.73 m2 (inclusive). Serum creatinine is tested and the eGFR is calculated by central laboratory using the Chronic Kidney Disease Epidemiology (CKD-EPI) equation.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 7
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 13
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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. The subject has a urine protein/creatinine ratio of >500 mg/g.
- •4. The subject has a clinically relevant history of allergy or signs or symptoms of severe hypersensitivity, which in the investigator’s judgment, will substantially increase the subject’s risk if he or she participates in the study.
- •5. In the opinion of the investigator, the subject has non-Fabry disease-related cause of end organ (renal, cardiovascular, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by renal measures.
- •6. The subject has a positive test result at screening for hepatitis B surface antigen with detectable hepatitis B viral DNA load, hepatitis C virus (HCV) antibody with confirmation by HCV ribonucleic acid polymerase chain reaction testing, or human immunodeficiency virus antibody.
- •7. The subject has received prior treatment with any of the following medications, with the exception of non-systemic use:
- •Chloroquine, • Amiodarone, • Monobenzone, • Gentamicin
- •8. The subject is pregnant or lactating.
- •9. The subject has a body mass index >35 kg/m2.
- •10. The subject is treated or has been treated with any investigational drug for indication other than Fabry disease within 30 days prior to study start.
- •11. The subject and/or the subject’s parent(s)/legal guardian is unable to understand the nature, scope, and possible consequences of the study.
- •12. 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 is otherwise unlikely to complete the study, as determined by the investigator.
研究者
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