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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
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
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
Where Is This Study? (2 UK sites)
Great Ormond Street Hospital for Children NHS Foundation Trust
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Manchester University NHS Foundation Trust
Manchester M13 9WL, United Kingdom
How to Get in Touch
Amicus Therapeutics Patient Advocacy
Sponsor contactCONTACT
