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New hope for fabry patients: Long-Term drug safety confirmed

NCT ID NCT04049760

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety of a drug called migalastat in 16 people over age 12 with Fabry disease, a rare genetic disorder. Participants had already completed an earlier study and continued taking migalastat to see if it was safe and how it affected kidney function. The goal was to manage the disease over time, not to cure it.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

16 people

The number who actually took part.

Started

Oct 2019

Finished

Nov 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male or female subjects diagnosed with Fabry disease \> 12 years of age who completed Study AT1001-020 * 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 * If of reproductive potential, both male and female subjects agreed 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: * Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was \< 60 mL/min/1.73 m2 * Subject had advanced kidney disease requiring dialysis or kidney transplantation * Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat * Subject anticipated starting gene therapy during the study period * Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study * Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol) * Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta) * Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat) * Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period * In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Lysosomal & Rare Disorders Research & Treatment Center

    Fairfax, Virginia, 22030, United States

  • Royal Free London NHS Foundation Trust

    London, United Kingdom

  • The Emory Clinic

    Atlanta, Georgia, 30322, United States

  • University of Minnesota Masonic Children's Hospital and Clinics

    Minneapolis, Minnesota, 55454, United States

  • University of South Florida

    Tampa, Florida, 33606, United States

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