Recruiting
Phase 3

Migalastat

Sponsor:

Amicus Therapeutics

Code:

NCT06904261

Conditions

Fabry Disease

Eligibility Criteria

Sex: All

Age: 2 - 11

Healthy Volunteers: Not accepted

Interventions

Migalastat HCl 20 mg

Study Details

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

Study ID

NCT06904261

Start date

Jan 8, 2026

Status verified date

Aug, 2026

Completion date

Dec, 2028

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 11

Healthy Volunteers: Not accepted

Inclusion Criteria

  • 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.

Exclusion Criteria

  • 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

Study Design

Enrollment

8 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Migalastat HCl 20 mg Dispersible Tablets

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

Interventions

Migalastat HCl 20 mg

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

Primary outcome measure

  • Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug [ Time Frame: Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose) ]
  • Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Migalastat [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12 ]

Central Contacts and Locations

Central contacts

Amicus Therapeutics Patient Advocacy

609-662-2000patientadvocacy@amicusrx.com

Locations

Emory Genetics

Recruiting

Atlanta, Georgia, United States, 30322

University of Minnesota Masonic Children's Hospital

Recruiting

Minneapolis, Minnesota, United States, 55455

Atrium Health Levine Children's Hospital

Recruiting

Charlotte, North Carolina, United States, 28203

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

UPMC Children's Hospital of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Recruiting

Fairfax, Virginia, United States, 22030

More Information

Sponsor

Amicus Therapeutics

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

  • migalastat
  • AT1001
  • Galafold
  • lysosomal disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Amicus Therapeutics on 2026-09-01.