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
Location: See all (15) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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)
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
• Subject (or legally authorized representative as applicable) is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information
• Subject has a GLA variant that is amenable to migalastat recorded in their medical records
• 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
• 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
• Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
• Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
• If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception
Locations
United States
Georgia
Emory University
RECRUITING
Atlanta
Ohio
The Cleveland Clinic
RECRUITING
Cleveland
Texas
Renal Disease Research Institute
WITHDRAWN
Dallas
Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc
RECRUITING
Fairfax
Other Locations
Australia
Royal Melbourne Hospital
RECRUITING
Parkville
Royal Perth Hospital
RECRUITING
Perth
France
Internal Medicine Unit Croix Saint Simon Hospital
WITHDRAWN
Paris
Japan
Shizuoka General Hospital
RECRUITING
Shizuoka
Osaka University Hospital
RECRUITING
Suita
Portugal
Centro Hospitalar e Universitário de Coimbra (CHUC)
RECRUITING
Coimbra
Spain
Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga
RECRUITING
Barcelona
Hospital Universitario Reina Sofia
WITHDRAWN
Córdoba
Hospital General Universitario de Elda
RECRUITING
Elda
Hospital General Universitario Gregorio Marañon
RECRUITING
Madrid
United Kingdom
Salford Royal Hospital
NOT_YET_RECRUITING
Salford
Contact Information
Primary
Amicus Therapeutics Patient Advocacy
patientadvocacy@amicusrx.com
609-662-2000
Time Frame
Start Date:2022-10-31
Estimated Completion Date:2026-12-31
Participants
Target number of participants:14
Treatments
Experimental: Cohort 1: Severe Renal Impairment
All subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat) at a dose regimen based on their eGFRMDRD result at Visit 1. Subjects will take 1 migalastat capsule orally with water either every 4 or 7 days.
Experimental: Cohort 2: End-Stage Renal Disease
All hemodialysis subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat). Subjects will take 1 migalastat capsule orally with water every other week.