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

NCT04049760 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: AT1001-0362019-000222-21
• Study Type: interventional
• Target: 16
• Locations: 2 countries
• Sites: 6

Brief 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

Conditions

Fabry Disease

Keywords

Lysosomal disease; migalastat

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug

  Number of subjects with TEAE, SAE, and AE leading to discontinuation during the study period

  Entire study

Secondary Outcomes (8)

• Change From Baseline to Month 24 in Estimated Glomerular Filtration Rate (eGFR)

  Baseline, Month 24

• Change From Baseline to Month 24 in Urine Protein Levels

  Baseline, Month 24

• Change From Baseline to Month 24 in Urine Albumin

  Baseline, Month 24

• Change From Baseline to Month 24 in Left Ventricular Mass Index (LVMi)

  Baseline, Month 24

• Change From Baseline to Month 24 in Pediatric and Quality of Life Inventory™ (PedsQL™) Scores

  Baseline, Month 24

Study Arms (1)

migalastat HCl 150 mg

EXPERIMENTAL

One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.

Drug: migalastat HCl 150 mg

Interventions

migalastat HCl 150 mg DRUG

migalastat HCl 150 mg capsule

Also known as: migalastat, AT1001

migalastat HCl 150 mg

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (6)

University of South Florida

Tampa, Florida, 33606, United States

Location

The Emory Clinic

Atlanta, Georgia, 30322, United States

Location

University of Minnesota Masonic Children's Hospital and Clinics

Minneapolis, Minnesota, 55454, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Lysosomal & Rare Disorders Research & Treatment Center

Fairfax, Virginia, 22030, United States

Location

Royal Free London NHS Foundation Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

migalastat; larazotide acetate

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Medical Information
• Organization: Amicus Therapeutics, Inc.

MedInfo@amicusrx.com

001 609-662-2000

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2019
• First Posted: August 8, 2019
• Study Start: October 11, 2019
• Primary Completion: November 29, 2024
• Study Completion: November 29, 2024
• Last Updated: February 5, 2026
• Results First Posted: June 17, 2025

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (5); GB (1)