A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: AT1001-033
• Study Type: interventional
• Target: 8
• Locations: 5 countries
• Sites: 11

Brief Summary

An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.

Conditions

Fabry Disease

Keywords

migalastat; AT1001; Galafold; lysosomal disease

Outcome Measures

Primary Outcomes (4)

• Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug

  Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)

• Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Migalastat

  0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12

• Pharmacokinetics (PK): Minimum Observed Plasma Concentration (Cmin) of Migalastat

  0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12

• Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) of Migalastat

  0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12

Secondary Outcomes (9)

• Pharmacodynamic: Change in plasma levels of lyso-Gb3 and its analogs from baseline

  Baseline to Months 3, 6, and 12/ET

• Efficacy: Change in eGFR from baseline

  Baseline to Months 1, 3, 6, and 12/ET

• Efficacy: Change in urine protein and albumin/microalbumin levels from baseline

  Baseline to Months 3, 6, and 12/ET

• Efficacy: Change in Left Ventricular Mass Index (LVMi) from baseline

  Baseline to Month 12/ET

• Efficacy: Change in FABPRO-GI And Pain Scores from baseline

  Baseline to Month 12/ET

Study Arms (1)

Migalastat HCl 20 mg Dispersible Tablets

EXPERIMENTAL

Migalastat will be administered every other day (QOD). The initial dose will be based on body weight at baseline.

Drug: Migalastat HCl 20 mg

Interventions

Migalastat HCl 20 mg DRUG

Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.

Also known as: AT1001, Galafold

Migalastat HCl 20 mg Dispersible Tablets

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female subjects, diagnosed with Fabry disease who are between ages 2 and \< 12 years at randomization (subjects aged 11 years must have birthdays \> 30 days after randomization)
• 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.
• Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
• Subject has not received ERT (eg, Replagal® \[agalsidase alfa\] or Fabrazyme® \[agalsidase beta\]) for at least 14 days prior to Baseline visit.
• Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
• If of reproductive potential, both male and female subjects agree 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:

• Has moderate or severe renal impairment (eGFR \< 60 mL/min/1.73 m2 at Visit 1 \[screening\]).
• Has advanced kidney disease requiring dialysis or kidney transplantation.
• History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
• Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
• Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
• Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
• Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
• Pregnant or breastfeeding
• Otherwise unsuitable for the study in the opinion of the investigator

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (11)

Emory Genetics

Atlanta, Georgia, 30322, United States

RECRUITING

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, 55455, United States

NOT YET RECRUITING

Atrium Health Levine Children's Hospital

Charlotte, North Carolina, 28203, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

NOT YET RECRUITING

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, 22030, United States

RECRUITING

Universitair Ziekenhuis (UZ) Leuven

Leuven, Vlaams-Brabant, 3000, Belgium

NOT YET RECRUITING

Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie

Münster, North Rhine-Westphalia, 48149, Germany

RECRUITING

Hospital Universitario de la Paz

Madrid, Madrid, 28046, Spain

RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3JH, United Kingdom

NOT YET RECRUITING

Manchester University NHS Foundation Trust

Manchester, M13 9WL, United Kingdom

RECRUITING

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

Central Study Contacts

Amicus Therapeutics Patient Advocacy

CONTACT

609-662-2000; patientadvocacy@amicusrx.com

Study Design

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

Study Record Dates

• First Submitted: March 13, 2025
• First Posted: April 1, 2025
• Study Start: January 8, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: April 24, 2026

Record last verified: 2025-06

Locations

GB (2); BE (1); US (6); ES (1); DE (1)