BridgeMD
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
EU CTIS ID: 2022-500488-10-00
What this study is testing
To characterize the pharmacokinetics (PK) of migalastat and confirm the proposed dosing regimens based on modeling and simulations in Fabry subjects with severe renal impairment (SRI) and end-stage renal disease (ESRD) on hemodialysis
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
- Subject is willing and able to provide written informed consent (or assent) and authorization for use and disclosure of Personal Health Information or research-related health information or subject has a legally-authorized representative who has provided written informed consent and authorization, and subject provides assent, if applicable.
- Subject has a GLA variant recorded in their medical records that is amenable to migalastat. • All subjects will have confirmatory GLA genotyping done at Visit 1. • For subjects without a known GLA variant, GLA genotyping results must be received prior to Visit 2. • For subjects with a GLA variant that has not yet been tested in the Migalastat Amenability Assay, amenability testing results must be received prior to Visit 2.
- Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 in his/her medical history within the last 3 months and has an eGFRMDRD value of < 30 mL/min/1.73 m2 at Visit 1 per the central laboratory or subject is on HD (standard or HDF).
- 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 (Visit 1).
- Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
You likely can't join if
- Subject has undergone or is scheduled to undergo kidney transplantation.
- Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
- Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
- Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta) or has been treated with these medications within 14 days before Visit 2.
- In France only, protected persons as defined by the Public Health Code.
- Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca within the 30 days before Visit 1.
See the full eligibility criteria
Who can join
- Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
- Subject is willing and able to provide written informed consent (or assent) and authorization for use and disclosure of Personal Health Information or research-related health information or subject has a legally-authorized representative who has provided written informed consent and authorization, and subject provides assent, if applicable.
- Subject has a GLA variant recorded in their medical records that is amenable to migalastat. • All subjects will have confirmatory GLA genotyping done at Visit 1. • For subjects without a known GLA variant, GLA genotyping results must be received prior to Visit 2. • For subjects with a GLA variant that has not yet been tested in the Migalastat Amenability Assay, amenability testing results must be received prior to Visit 2.
- Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 in his/her medical history within the last 3 months and has an eGFRMDRD value of < 30 mL/min/1.73 m2 at Visit 1 per the central laboratory or subject is on HD (standard or HDF).
- 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 (Visit 1).
- 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 subjects agree to use a medically accepted method of contraception during the study and for up to 30 days after the last dose of migalastat.
What rules you out
- Subject has undergone or is scheduled to undergo kidney transplantation.
- Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
- Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
- Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta) or has been treated with these medications within 14 days before Visit 2.
- In France only, protected persons as defined by the Public Health Code.
- Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca within the 30 days before Visit 1.
- Female subject is pregnant or breastfeeding.
- Subject is unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator.
- Subject is on peritoneal dialysis.
- Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days before Visit 1.
- Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study.
- Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1.
- Subject has clinically significant unstable cardiac disease in the opinion of the investigator (eg, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association Class III or IV congestive heart failure).
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Australia
- Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Australia; Japan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.