MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study (MALTA-FABRY)

  • End date
    Dec 31, 2023
  • participants needed
  • sponsor
    Wuerzburg University Hospital
Updated on 24 July 2022


This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.


Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Since 2016, chaperone therapy as a new therapeutic approach is available.

This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy.

All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided.

Condition Rare Diseases, Fabry Disease, Adherence, Medication, Quality of Life
Clinical Study IdentifierNCT03683966
SponsorWuerzburg University Hospital
Last Modified on24 July 2022


Yes No Not Sure

Inclusion Criteria

Fabry disease (genetically confirmed)
Signed informed consent
years and older

Exclusion Criteria

No informed consent
Withdrawal of informed consent
Clear my responses

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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