New hope for fabry patients: Long-Term drug safety confirmed
NCT ID NCT04049760
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.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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16 people
The number who actually took part.
- Started
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Oct 2019
- Finished
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Nov 2024
- Lead sponsor
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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
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12 to 17 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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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
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, 22030, United States
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Royal Free London NHS Foundation Trust
London, United Kingdom
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The Emory Clinic
Atlanta, Georgia, 30322, United States
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University of Minnesota Masonic Children's Hospital and Clinics
Minneapolis, Minnesota, 55454, United States
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University of South Florida
Tampa, Florida, 33606, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a new pill stop fatty buildup in fabry disease?
- Gene Therapy's lasting promise: can one infusion safely control fabry disease for years?
- Can early enzyme therapy save kidneys in fabry disease?
- Can continued lucerastat access help fabry patients?
- Can a single gene infusion rewrite the story of fabry disease?