A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055 · tracked via the Priya Life Science UK tracker

Sponsor

Amicus Therapeutics

Phase

Phase 3

Started

2022-10-31

Last updated

2026-07-06

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

migalastat HCl 150 mg

migalastat HCl 150 mg: migalastat HCl 150 mg capsule

Study 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

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: 1. Male or female subjects aged 18 years or older, diagnosed with Fabry disease. 2. 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 3. Subject has a GLA variant that is amenable to migalastat recorded in their medical records 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 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 Exclusion Criteria: 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 breast-feeding 12. Subject is unable to comply with study requirements 13. In France only, protected persons as defined by the Public Health Code

Primary outcome measure(s)

Maximum observed concentration (Cmax) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Time to maximum concentration (tmax) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Apparent terminal elimination half-life (t½) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK
Concentration at the end of a dosing interval at steady state (Ctrough) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Average plasma migalastat concentration over the dosing interval (Cavg) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Area under the concentration-time curve at steady state during the dosing interval (AUC0-τ) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Area under the concentration-time curve from zero time (pre-dose) extrapolated to infinite time (AUC0-∞) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Apparent plasma clearance (CL/F) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK
Apparent terminal phase volume of distribution (Vz/F) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Dialysis clearance (CLD) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Volume of dialysate collected during the interval (VD) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Mean migalastat concentration in dialysate (CD) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Amount recovered in dialysate (AeD) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Fraction of the dose recovered in dialysate (FeD) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Mean migalastat plasma concentration during the dialysis interval (P) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Mean inlet area under the curve (AUCinlet) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Mean outlet area under the curve (AUCoutlet) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Extraction ratio (ED) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Dialyzer blood flow (QD) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Cumulative amount excreted over all collection intervals (Ae0-τ) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Fraction of the dose recovered after the last measurable time point postdose (Fe0-τ) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.
Renal clearance (CLr) — Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations.

Trial sites (12)

Facility	City	Region
Emory University	Atlanta	Georgia
The Cleveland Clinic	Cleveland	Ohio
Lysosomal and Rare Disorders Research and Treatment Center, Inc	Fairfax	Virginia
Royal Melbourne Hospital	Parkville	Victoria
Royal Perth Hospital	Perth	Washington
Osaka University Hospital	Suita	Osaka
Shizuoka General Hospital	Shizuoka	Shizuoka
Centro Hospitalar e Universitário de Coimbra (CHUC)	Coimbra	Portugal
Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga	Barcelona	Spain
Hospital General Universitario de Elda	Elda	Spain
Hospital General Universitario Gregorio Marañon	Madrid	Spain
Salford Royal Hospital	Salford	England

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04020055 on ClinicalTrials.gov ↗ ← All trials in the UK