DRUG
migalastat HCl 150 mg
migalastat HCl 150 mg capsule
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Status
Active, not recruiting
Phase
Phase 3
Enrollment
14
Locations
12
Results
Not posted
Publications
0
Study summary
What the protocol is testing.
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)
Full 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 mL/min/1.73 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 HD/HDF 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 mL/min/1.73 m2) may be re-screened.
Interventions
Treatment arms and agents.
Timeline
From registration to results.
First posted
Jul 15, 2019
Study start
Oct 31, 2022
Primary completion
Dec 31, 2026
Study completion
Dec 31, 2026
Results posted
Not reported
Registry updated
Jul 6, 2026
Outcomes
What the study measures.
Primary outcomes
Maximum observed concentration (Cmax)
Time frame · 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)
Time frame · 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)
Time frame · 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)
Time frame · 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)
Time frame · 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)
Time frame · 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-∞)
Time frame · 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)
Time frame · Baseline through Month 12
To characterize the pharmacokinetics (PK) of migalastat and validate the population PK
Secondary outcomes
Adverse events (AEs)
Time frame · Baseline through Month 12
To assess the safety and tolerability of migalastat in Fabry subjects with severe renal impairment and end stage renal disease.
Change from baseline in estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease equation (eGFR MDRD)
Time frame · Baseline through Month 12
To assess the safety and tolerability of migalastat in Fabry subjects with severe renal impairment and end stage renal disease
Change from baseline eGFR based on the Chronic Kidney Disease Epidemiology Collaboration equation (eGFRCKD-EPI)
Time frame · Baseline through Month 12
To assess the safety and tolerability of migalastat in Fabry subjects with severe renal impairment and end stage renal disease
Change from baseline in plasma globotriaosylsphingosine (lyso-Gb3)
Time frame · Baseline through Month 12
To evaluate the pharmacodynamics (PD) of migalastat in subjects with Fabry disease and severe renal impairment
Eligibility
Who can take part.
- Minimum age
- 18 Years
- Maximum age
- Not reported
- Sex
- ALL
- Healthy volunteers
- No
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
Study locations
12 registered sites.
Australia · Japan · Portugal · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Emory University
Atlanta, Georgia, United States
The Cleveland Clinic
Cleveland, Ohio, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc
Fairfax, Virginia, United States
Royal Melbourne Hospital
Parkville, Victoria, Australia
Royal Perth Hospital
Perth, Washington, Australia
Osaka University Hospital
Suita, Osaka, Japan
Shizuoka General Hospital
Shizuoka, Shizuoka, Japan
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, United Kingdom
Publications
Results and literature.
No PMID-linked publications were present in this registry snapshot.
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