A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease (NCT04020055) | Clinical Trial Compass
Active β Not RecruitingPhase 3
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
United States, Australia14 participantsStarted 2022-10-31
Plain-language 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)
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
β. Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
β. 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
β. Subject has a GLA variant that is amenable to migalastat recorded in their medical records
β. 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
β. 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
β. Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
β. Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
β. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception
Exclusion criteria
β. Subject has undergone kidney transplantation
β. Subject is on peritoneal dialysis
β. Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days
What they're measuring
1
Maximum observed concentration (Cmax)
Timeframe: Baseline through Month 12
2
Time to maximum concentration (tmax)
Timeframe: Baseline through Month 12
3
Apparent terminal elimination half-life (tΒ½)
Timeframe: Baseline through Month 12
4
Concentration at the end of a dosing interval at steady state (Ctrough)
Timeframe: Baseline through Month 12
5
Average plasma migalastat concentration over the dosing interval (Cavg)
Timeframe: Baseline through Month 12
6
Area under the concentration-time curve at steady state during the dosing interval (AUC0-Ο)
Timeframe: Baseline through Month 12
7
Area under the concentration-time curve from zero time (pre-dose) extrapolated to infinite time (AUC0-β)