Ignite Creation Date:
2024-05-06 @ 1:27 PM
Last Modification Date:
2024-10-26 @ 1:14 PM
Study NCT ID:
NCT04020055
Status:
RECRUITING
Last Update Posted:
2024-07-01
First Post:
2019-06-24
Brief Title:
A Study to Evaluate Migalastat in Fabry Subjects with Amenable GLA Variant and Renal Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
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
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 SRI or End Stage Renal Disease ESRD
Detailed Description:
This is an open-label non-comparative study for subjects with Fabry disease who have an estimated glomerular filtration rate eGFR based on the Modification of Diet in Renal Disease equation eGFRMDRD value of 30 mLmin173 m2 Subjects may have had previous exposure to migalastat either commercially or as a participant in a previous migalastat study
Two distinct populations of subjects with Fabry disease and renal impairment will be enrolled into this study
Cohort 1 Subjects with SRI not receiving any type of dialysis treatment
Cohort 2 ESRD subjects who are receiving hemodialysis treatment either standard hemodialysis HD or hemodiafiltration HDF Only subjects who can receive HDHDF at the study clinic or at an affiliated center where the Investigator already has oversight should be enrolled into Cohort 2
Subjects entering into this study will undergo screening Visit 1 to confirm enrollment eligibility including confirmatory GLA genotyping Subjects who meet eligibility criteria will have a Baseline Visit Visit 2 within 30 days of screening Subjects who do not meet eligibility criteria eg subjects with an eGFR 30 mLmin173 m2 may be re-screened
Two distinct populations of subjects with Fabry disease and renal impairment will be enrolled into this study
Cohort 1 Subjects with SRI not receiving any type of dialysis treatment
Cohort 2 ESRD subjects who are receiving hemodialysis treatment either standard hemodialysis HD or hemodiafiltration HDF Only subjects who can receive HDHDF at the study clinic or at an affiliated center where the Investigator already has oversight should be enrolled into Cohort 2
Subjects entering into this study will undergo screening Visit 1 to confirm enrollment eligibility including confirmatory GLA genotyping Subjects who meet eligibility criteria will have a Baseline Visit Visit 2 within 30 days of screening Subjects who do not meet eligibility criteria eg subjects with an eGFR 30 mLmin173 m2 may be re-screened
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None