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

AT1001-025

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)

2022-10-31

2024-12-31

2025-12-31

Recruiting

Early phase I

14

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 14, 'type': 'ESTIMATED'}}

2024-04-01