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NCT04020055Phase 3ACTIVE NOT RECRUITING

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

Study 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)

Conditions
Fabry Disease
Interventions / Drugs
migalastat HCl 150 mg
View on ClinicalTrials.gov ↗
Sponsor
FOLD
Amicus Therapeutics
Rare Disease
C57/100
Study Details
Enrollment14 participants
Primary Completion—
Lead SponsorAmicus Therapeutics
Data Retrieved2026-04-11 04:14:12.640583+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 14 participants
Sponsor Catalyst Profile

Amicus Therapeutics (FOLD) is the sponsoring company for this trial. BiotechSign currently grades this company C (57/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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