Phase 3 N=16 Treatment

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Fabry Disease

Bottom Line

View on ClinicalTrials.gov: NCT04049760 ↗

Enrolled (actual)

Serious AEs

6.3%

Results posted

Jun 2025

Primary outcome: Primary: Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug — 13; 1; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: migalastat HCl 150 mg (Drug)
Age: Pediatric · 12+ yrs
Sex: All
Sponsor: Amicus Therapeutics
Primary completion: Nov 2024

Outcome Measures

Outcome	Result	p-value
PRIMARY Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug	13; 1; 0	—
SECONDARY Change From Baseline to Month 24 in Estimated Glomerular Filtration Rate (eGFR)	-27.6	—
SECONDARY Change From Baseline to Month 24 in Urine Protein Levels	38.6	—
SECONDARY Change From Baseline to Month 24 in Urine Albumin	-12.7	—
SECONDARY Change From Baseline to Month 24 in Left Ventricular Mass Index (LVMi)	4.75; 6.01	—
SECONDARY Change From Baseline to Month 24 in Pediatric and Quality of Life Inventory™ (PedsQL™) Scores	0; 0; -1.1	—
SECONDARY Change From Baseline to Month 24 in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ) Score for Pain Intensity	-1.3	—
SECONDARY Number of Subjects Who Experienced Sudden Onset of Pain As Assessed Using the Fabry-Specific Health and Pain Questionnaire (FPHPQ)	3; 4; 0; 1	—
SECONDARY Change From Baseline to Month 24 in Plasma Levels of Lyso-Gb3	0.05	—

Summary

This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants

Eligibility Criteria

Inclusion Criteria

Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
If of reproductive potential, both male and female subjects agreed to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat

Exclusion Criteria

Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was < 60 mL/min/1.73 m2
Subject had advanced kidney disease requiring dialysis or kidney transplantation
Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat
Subject anticipated starting gene therapy during the study period
Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study
Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta)
Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period
In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04049760). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.