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

July 6, 2022 updated by: PD Dr. Peter Nordbeck, Wuerzburg University Hospital

A Structured Survey Among Fabry Patients With a Focus on Adherence to Therapy, Quality of Life and Pain Control

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

Study Overview

Status

Recruiting

Conditions

Detailed 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.

Study Type

Observational

Enrollment (Anticipated)

30

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Peter Nordbeck, MD, PhD
  • Phone Number: 004993120139181
  • Email: nordbeck_p@ukw.de

Study Contact Backup

Study Locations

  • Germany
    • Bayern
      • Würzburg, Bayern, Germany, 97080
        • Recruiting
        • Wuerzburg University Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The study population includes every patient with Fabry disease and therapy who is seen in the FAZiT Wuerzburg. Limitation is a therapy with migalastat.

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • No informed consent
  • Withdrawal of informed consent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adherence to oral therapy with migalastat according to Medication Assessment Questionnaire
Time Frame: From date of inclusion up to104 weeks (2 years)
Pharmacological adherence according to adapted and translated 'Medication Assessment Questionnaire'.
From date of inclusion up to104 weeks (2 years)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quality of Life according to SF-36 and Wuerzburg pain questionnaire
Time Frame: From date of inclusion up to104 weeks (2 years)

Change of quality of life under migalastat therapy according to SF-36 and Wuerzburg pain questionnaire. Short Form (36), abbreviated SF-36, is a disease-specific measurement tool for increasing the health-related quality of life. The SF-36 is composed of eight scale-valued domains that correspond to the weighted sums of answers in each section. The range of values of each scale is 0-100 under the assumption that each question has the same weight. A score of 0 is the worst outcome (maximum disability) and a score of 100 is the best outcome (no disability).

The eight domains of the SF-36 are vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning and mental health.
From date of inclusion up to104 weeks (2 years)

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pain according to SF-36 and Wuerzburg pain questionnaire
Time Frame: From date of inclusion up to104 weeks (2 years)

Change of pain under migalastat therapy according to SF-36 and Wuerzburg pain questionnaire.

The Wuerzburg pain questionnaire was created by Üçeyler et al. In this questionnaire, the patient's pain phenotype is characterized with questions about the presence of 1) permanent pain, 2) pain attacks, 3) pain crisis, and 4) evoked pain (by touching a cold or warm object or by pressure) in childhood and/or adulthood with the response options Yes, No, or Don't know. Other criteria can be found in the publication (Üçeyler N, Ganendiran S, Kramer D, Sommer C. Characterization of pain in fabry disease. Clin J Pain. 2014 Oct;30(10):915-20.).
From date of inclusion up to104 weeks (2 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Wuerzburg University Hospital

Collaborators

Charite University, Berlin, Germany
University Hospital Muenster
Health Care Center Dr. Markus Cybulla, Muellheim

Investigators

  • Principal Investigator: Peter Nordbeck, MD, PhD, Wuerzburg University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 27, 2017

Primary Completion (Anticipated)

December 31, 2023

Study Completion (Anticipated)

December 31, 2023

Study Registration Dates

First Submitted

September 3, 2018

First Submitted That Met QC Criteria

September 24, 2018

First Posted (Actual)

September 25, 2018

Study Record Updates

Last Update Posted (Actual)

July 11, 2022

Last Update Submitted That Met QC Criteria

July 6, 2022

Last Verified

July 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AT-IIP-2018

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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