An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-stage Renal Disease Treated with Hemodialysis

Koh KyuBum0 个研究点目标入组 14 人开始时间: 2022年12月2日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•Subjects eligible for enrollment in the study must meet all of the following criteria:
•1. Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
•2. Subject is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information.
•3. Subject has a GLA variant recorded in their medical records that is amenable to migalastat.
•4. Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 within the last 3 months and has an eGFRMDRD value of < 30 mL/min/1.73 m2 at Visit 1 or subject is on HD (standard or HDF).
•5. Subjects with ESRD have been on a stable 2- or 3-times a week HD (standard or HDF) regimen for at least 2 months prior to the screening visit.
•6. Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
•7. Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
•8. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception

•Subjects meeting any of the following criteria are not eligible to be enrolled in the study:
•1. Subject has undergone kidney transplantation.
•2. Subject is on peritoneal dialysis.
•3. Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days.
•4. Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study.
•5. Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction.
•6. Subject has clinically significant unstable cardiac disease.
•7. Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements.
•8. Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
•9. Subject requires concurrent treatment with Glyset (miglitol), Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta).
•10. Subject requires concurrent treatment with Zavesca (miglustat) or has been treated with Zavesca.
•11. Female subject is pregnant or breastfeeding.
•12. Subject is unable to comply with study requirements.

发起方

Koh KyuBum