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Looking for participantsPhase3

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

Sponsor: Amicus Therapeutics

NCT ID: NCT06904261

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Migalastat HCl 20 mg (drug)
How long the study runs
Study runs about 35 months (dates as stated)
About the drug or intervention
Migalastat HCl 20 mg — drug: Migalastat will be supplied as 20-mg dispersible tablets.
Patient visit burden
Not specified by the sponsor

In plain English

This study, run by Amicus Therapeutics, is testing a medicine called migalastat in children aged 2 to under 12 who have Fabry disease. Fabry disease is a rare inherited condition, and children in this study must have a change (variant) in a gene called GLA that is expected to respond to migalastat.

Who can take part

  • Children aged 2 to under 12 with Fabry disease (those aged 11 must have a birthday more than 30 days after joining)
  • A parent or legal representative can give written permission, and the child agrees where this applies
  • A GLA gene variant that is 'amenable' (expected to respond to migalastat) is recorded in their medical notes before Visit 2
  • Has not had enzyme replacement therapy (ERT), such as Replagal or Fabrazyme, for at least 14 days before the baseline visit
  • Has at least one known complication of Fabry disease, such as an abnormal test result, sign or symptom
  • If able to have children, agrees to use an accepted method of contraception during the study and for up to 30 days after the last dose

Who may not be able to

  • Moderate or severe kidney problems (eGFR below 60 mL/min/1.73 m2 at screening)
  • Advanced kidney disease needing dialysis or a kidney transplant
  • Allergy or sensitivity to migalastat, its ingredients, or similar medicines called iminosugars (such as miglustat or miglitol)
  • Has taken any experimental drug, biologic or device within 30 days or 5 half-lives (whichever is longer) before screening
  • Has had gene therapy at any time, or plans to start it during the study
  • Needs treatment with Glyset (miglitol) or Zavesca (miglustat) within 6 months before screening or during the study
  • Another illness or condition that, in the researcher's view, would make taking part unsafe or too difficult
  • Pregnant or breastfeeding
  • Any other reason the researcher thinks the child is not suitable for the study

What taking part involves

  • • Not stated — ask the trial team
  • • Children must stop enzyme replacement therapy (ERT) at least 14 days before the baseline visit to take part

Time commitment: Number of visits, study length and what taking part involves day to day are not stated — ask the trial team.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
2 Years to 11 Years
Who
All
Number of participants
8
Started
2026-01-08
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for fabry disease
  • • Phase3 - 8 participants
  • • 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

Who can take part?

  • • Ages 2 Years to 11 Years
  • • Diagnosed with fabry disease

Where?

  • • London - Great Ormond Street Hospital for Children NHS Foundation Trust
  • • Manchester - Manchester University NHS Foundation Trust

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

More detail

This is a Phase 3b, 2-stage, open-label, uncontrolled, multicenter study to evaluate the safety, PK, PD, and efficacy of 12 months of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants. Subjects must be either naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days before Baseline visit. The study will consist of 2 treatment stages followed by an open-label extension (OLE). Stage 1 will be a treatment period of approximately 3 months (12 weeks); Stage 2 will be a treatment period of 9 months. There will be no break in treatment between Stages 1 and 2. There will be a 30-day (untreated) safety follow-up period for subjects who discontinue treatment at any time. Subjects will be randomly assigned 1:1:1 to 1 of 3 PK sampling groups using interactive response technology (IRT). Four blood samples for the determination of migalastat concentrations in plasma will be collected in one 24-hour period between Day 15 and Day 30 and at Month 6, and 1 PK (trough) sample will be collected at Month 6 and again at Month 12.

Fabry Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 2 Years - 11 Years
  • Who can join: All genders

Biomarkers mentioned

eGFR

Treatment history

Treatments you must have had:

  • ✓ birthdays \> 30 days after randomization)
  • ✓ Glyset (miglitol) or Zavesca (miglustat), within 6 months

What the study is looking for

  • ✓Inclusion Criteria
  • ✓Male or female subjects, diagnosed with Fabry disease who are between ages 2 and \< 12 years at randomization...
  • ✓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...
  • ✓Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of...
See the full criteria
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

Where Is This Study? (2 UK sites)

Great Ormond Street Hospital for Children NHS Foundation Trust

London WC1N 3JH, United Kingdom

NOT_YET_RECRUITING
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

Manchester University NHS Foundation Trust

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

How to Get in Touch

Amicus Therapeutics Patient Advocacy

Sponsor contact

CONTACT

609-662-2000 patientadvocacy@amicusrx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-08