Active — Not RecruitingPhase 3

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

United States, Australia, Japan14 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 range

18 Years

Sex

ALL

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

. 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

Exclusion criteria

. Subject has undergone kidney transplantation

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Maximum observed concentration (Cmax)

Timeframe: Baseline through Month 12

Time to maximum concentration (tmax)

Timeframe: Baseline through Month 12

Apparent terminal elimination half-life (t½)

Timeframe: Baseline through Month 12

Concentration at the end of a dosing interval at steady state (Ctrough)

Timeframe: Baseline through Month 12

Average plasma migalastat concentration over the dosing interval (Cavg)

Timeframe: Baseline through Month 12

Area under the concentration-time curve at steady state during the dosing interval (AUC0-τ)

Timeframe: Baseline through Month 12

Area under the concentration-time curve from zero time (pre-dose) extrapolated to infinite time (AUC0-∞)

Timeframe: Baseline through Month 12

Trial details

NCT IDNCT04020055

SponsorAmicus Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-12-31

View on ClinicalTrials.gov More Fabry Disease trials

Who can participate

Age range

18 Years

Sex

ALL

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

. 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

Exclusion criteria

. Subject has undergone kidney transplantation

What they're measuring

Maximum observed concentration (Cmax)

Timeframe: Baseline through Month 12

Time to maximum concentration (tmax)

Timeframe: Baseline through Month 12

Apparent terminal elimination half-life (t½)

Timeframe: Baseline through Month 12

Concentration at the end of a dosing interval at steady state (Ctrough)

Timeframe: Baseline through Month 12

Average plasma migalastat concentration over the dosing interval (Cavg)

Timeframe: Baseline through Month 12

Area under the concentration-time curve at steady state during the dosing interval (AUC0-τ)

Timeframe: Baseline through Month 12

Area under the concentration-time curve from zero time (pre-dose) extrapolated to infinite time (AUC0-∞)

Timeframe: Baseline through Month 12