A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment

• Status: Not yet recruiting
• Trial end date: 2025-12-31
• Target enrollment: 0
• Participant gender: All

Summary

This is an Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics
• Treatments: 1-Deoxynojirimycin

Criteria

Inclusion Criteria:

1. Male or female subjects aged 16 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

5. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception

Exclusion Criteria:

1. Subject has an eGFR of ≥ 30 mL/min/1.73 m2 at Visit 1 per the central laboratory.

2. Subject has undergone kidney transplantation or is currently on dialysis, or is planned to start dialysis within 3 months

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 and 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