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临床试验/CTRI/2021/12/038690
CTRI/2021/12/038690进行中（未招募）4 期

A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase alfa (r-DNA origin) (Replagalâ?¢) in Indian Children and Adults With Fabry Disease - NA

Shire Biotech India Pvt Ltd0 个研究点目标入组 0 人开始时间: 待定最近更新:
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试验速览

阶段
4 期
状态
进行中（未招募）
发起方
研究概览入排标准研究者记录与标识符相似试验

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

入选标准

  • •1. Male and female Replagal naïve subjects (and who are not part of any other program at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Fabry disease.
  • •2. Subjects who have documented confirmed diagnosis of Fabry disease based on proof of gene mutation: α-galactosidase A gene compatible with Fabry disease and/or a deficiency of α-galactosidase A ( <4.0 nmol/mL/hour in plasma or serum or <8% of average mean normal in leukocytes and sequencing of GLA gene for females).
  • •3. Subject must have any clinical manifestations of Fabry disease based on investigatorâ??s discretion.
  • •4. Subject/LAR/guardian is able to understand and willing to give written informed consent before performing any study specific procedures and willing to adhere to protocol requirements.
  • •5. Female subjects of childbearing potential (eg, nonsterilised, premenopausal female subjects) must have a documented negative pregnancy test prior to administration of the first dose of Replagal in this study. In addition, all female subjects of childbearing potential must use a medically accepted form of contraception throughout the study, ie, either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.
  • •6. Male subject who is nonsterilised and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (eg, condom with or without spermicide) from signing of informed consent throughout the duration of the study.
  • •Note: Female subjects not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (eg, defined as at least 1 year since last regular menses with an appropriate clinical profile [ie, age appropriate, history of vasomotor symptoms]

排除标准

  • •1. Subject who have received Replagal.
  • •2. Subjects with poorly controlled hypertension as per investigatorâ??s discretion.
  • •3. Subjects with chronic kidney disease (CKD) with estimated Glomerular Filtration rate less than 15 mL/min/1.73 m2 and who had/will have kidney transplantation or are currently on dialysis.
  • •4. Subjects with any serious hepatic disorder who had abnormal hepatic function test values at screening (when either ALT or AST level exceeded the value three times the upper limit of normal [ULN] and total bilirubin 1.5 times as high as the ULN); and deemed as clinically significant by investigstor for hematology and biochemistry. These abnormal laboratory values could be discussed with medical monitor before excluding the subject.
  • •5. If female, the subject is pregnant or lactating or intending to become pregnant before participating in this study, during the study; or intending to donate ova during such time period.
  • •6. Subject/LAR/guardian is unable to understand the nature, scope, and possible consequences of the study.
  • •7. 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 safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
  • •8. If male, the subject intends to donate sperm during the course of this study.
  • •9. Subjects who had participated in any other investigational drug study within the past 4 weeks prior to screening.
  • •10. Any subject deemed as unfit for this trial, as per investigatorâ??s clinical judgment.

研究者

发起方
Shire Biotech India Pvt Ltd

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