Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 3INTERVENTIONAL

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

Q: What is the NCT06904261 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Q: Who can participate in NCT06904261?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

Q: How do I contact the trial site for NCT06904261?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Q: Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Q: Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

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

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants (NCT06904261) is a Phase 3 interventional studying Fabry Disease, sponsored by Amicus Therapeutics. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 3 trials confirm efficacy and safety in large patient groups (often 300–3,000+) and form the evidence base for an FDA approval submission. For Fabry Disease, Phase 3 studies typically randomize participants between the investigational treatment and either a placebo or current standard of care. A successful Phase 3 result is the threshold most treatments need to clear before regulatory approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 8 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

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 willing to sign a consent form 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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

Treatments Being Tested

DRUG

Migalastat HCl 20 mg

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

Locations (11)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Emory Genetics

Atlanta, Georgia, United States

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, United States

Atrium Health Levine Children's Hospital

Charlotte, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, United States

Universitair Ziekenhuis (UZ) Leuven

Leuven, Vlaams-Brabant, Belgium

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

Münster, North Rhine-Westphalia, Germany

Hospital Universitario de la Paz

Madrid, Madrid, Spain

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Manchester University NHS Foundation Trust

Manchester, United Kingdom

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06904261), the sponsor (Amicus Therapeutics), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06904261 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06904261?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06904261?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06904261. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06904261. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.