Tests migalastat in young children with Fabry disease
Tests the drug migalastat in children aged 2 to 11 with Fabry disease.
Plain-language title generated from the registry record
Registry title: A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Key facts
- Where
- Madrid, SpainSee the site
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children aged 2 to under 12 years with Fabry disease and a GLA gene variant that responds to migalastat.
- Age
- 2 to under 12 years (children aged 11 must have a birthday more than 30 days after randomization)
- Sex
- Boys and girls can join.
- Disease stage
- Fabry disease with at least 1 documented complication, such as a past or current lab abnormality or symptom.
- Earlier treatment
- No enzyme replacement therapy (ERT, such as Replagal or Fabrazyme) for at least 14 days before the baseline visit.
- Biomarker or test result
- A GLA variant in the medical record that is amenable to migalastat.
Main reasons for not taking part
- Moderate or severe kidney impairment (eGFR < 60 mL/min/1.73 m2)
- Kidney disease needing dialysis or a kidney transplant
- Allergy to migalastat or other iminosugars (for example miglustat, miglitol)
- Any gene therapy, now or planned during the study
- Pregnant or breastfeeding
Read by machine on 12 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 11 years old?
Source: NCT06904261, who can take part
Where in Spain
- Hospital Universitario de la PazMadrid, SpainContact: Amicus Therapeutics Patient AdvocacyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Amicus Therapeutics
- Trial code
- NCT06904261
- Record updated
- 22 October 2026
- Original description
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.Read full original descriptionShow less
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants
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.
Inclusion: 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: 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 advanced kidney disease requiring dialysis or kidney transplantation; History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol); 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; Has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening]); Pregnant or breastfeeding; Otherwise unsuitable for the study in the opinion of the investigator.