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

  • STATUS
    Recruiting
  • End date
    Dec 31, 2022
  • participants needed
    30
  • sponsor
    Wuerzburg University Hospital
Updated on 14 February 2022
migalastat

Summary

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

Description

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.

Details
Condition Rare Diseases, Fabry Disease, Adherence, Medication, Quality of Life
Clinical Study IdentifierNCT03683966
SponsorWuerzburg University Hospital
Last Modified on14 February 2022

Eligibility

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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Step 1 Connect with a study center
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  • You can expect the study team to contact you via email or phone in the next few days.
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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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