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Rare DiseasesMALTA-FABRYNCT03683966

MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study

An observational study of Rare Diseases and Fabry Disease, run by Wuerzburg University Hospital at 1 site in Germany.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

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

Who is it for?
Ages 18 years and older
How many people
30 participants
Where
1 siteGermany
Study dates
27 Oct 2017to 31 Dec 2023
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • Adult, Older adult

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.

Who may be able to join

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

Who cannot join

  • No informed consent
  • Withdrawal of informed consent

Trial locations and contacts

1 site in 1 country.

Peter Nordbeck, MD, PhD contact

004993120139181

nordbeck_p@ukw.de

Jonas Muentze, MD contact

004993120139958

muentze_j@ukw.de

Germany1 site, 1 recruiting

  • Wuerzburg University Hospital

    Würzburg, Bavaria, 97080

    Irina Schumacher, 004993120139714, schumacher_i@ukw.de

    Recruiting

Other trials in Germany

About this study

Official title: 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.

Read the 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.

Keywords
Fabry, Chaperone therapy, Migalastat, Pharmacological adherence
Medical subject headings
Rare Diseases, Fabry Disease, Medication Adherence

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adherence to oral therapy with migalastat according to Medication Assessment Questionnaire

    Pharmacological adherence according to adapted and translated 'Medication Assessment Questionnaire'.

    Measured: From date of inclusion up to104 weeks (2 years)

Secondary outcomes (1)

  1. Quality of Life according to SF-36 and Wuerzburg pain questionnaire

    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.

    Measured: From date of inclusion up to104 weeks (2 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Sep 2018
  2. Study start27 Oct 2017
  3. Primary completion31 Dec 2023 (estimated)
  4. Study completion31 Dec 2023 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03683966
Sponsor's protocol ID
AT-IIP-2018
Acronym
MALTA-FABRY
Status last verified
Jul 2022
First submitted
3 Sep 2018
Last updated
11 Jul 2022
Results posted
No

Publications

  • Muntze J, Salinger T, Gensler D, Wanner C, Nordbeck P. Treatment of hypertrophic cardiomyopathy caused by cardiospecific variants of Fabry disease with chaperone therapy. Eur Heart J. 2018 May 21;39(20):1861-1862. doi: 10.1093/eurheartj/ehy072. No abstract available.

    PubMed 29452394

  • Oder D, Liu D, Hu K, Uceyler N, Salinger T, Muntze J, Lorenz K, Kandolf R, Grone HJ, Sommer C, Ertl G, Wanner C, Nordbeck P. alpha-Galactosidase A Genotype N215S Induces a Specific Cardiac Variant of Fabry Disease. Circ Cardiovasc Genet. 2017 Oct;10(5):e001691. doi: 10.1161/CIRCGENETICS.116.001691.

    PubMed 29018006

  • Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198.

    PubMed 27509102

  • Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T, Olivotto I, Sakai N, Deegan P, Dimmock D, Eyskens F, Germain DP, Goker-Alpan O, Hachulla E, Jovanovic A, Lourenco CM, Narita I, Thomas M, Wilcox WR, Bichet DG, Schiffmann R, Ludington E, Viereck C, Kirk J, Yu J, Johnson F, Boudes P, Benjamin ER, Lockhart DJ, Barlow C, Skuban N, Castelli JP, Barth J, Feldt-Rasmussen U. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10.

    PubMed 27834756

  • Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.

    PubMed 27657681

  • Lenders M, Nordbeck P, Kurschat C, Karabul N, Kaufeld J, Hennermann JB, Patten M, Cybulla M, Muntze J, Uceyler N, Liu D, Das AM, Sommer C, Pogoda C, Reiermann S, Duning T, Gaedeke J, Stumpfe K, Blaschke D, Brand SM, Mann WA, Kampmann C, Muschol N, Canaan-Kuhl S, Brand E. Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS). Clin Pharmacol Ther. 2020 Aug;108(2):326-337. doi: 10.1002/cpt.1832. Epub 2020 Apr 27.

    PubMed 32198894

  • Muntze J, Gensler D, Maniuc O, Liu D, Cairns T, Oder D, Hu K, Lorenz K, Frantz S, Wanner C, Nordbeck P. Oral Chaperone Therapy Migalastat for Treating Fabry Disease: Enzymatic Response and Serum Biomarker Changes After 1 Year. Clin Pharmacol Ther. 2019 May;105(5):1224-1233. doi: 10.1002/cpt.1321. Epub 2019 Jan 13.

    PubMed 30506669

Questions about this trial

What is this clinical trial studying?

An observational study of Rare Diseases and Fabry Disease, run by Wuerzburg University Hospital at 1 site in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 11 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Peter Nordbeck, MD, PhD (004993120139181, nordbeck_p@ukw.de), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adherence to oral therapy with migalastat according to Medication Assessment Questionnaire (time frame: From date of inclusion up to104 weeks (2 years)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03683966, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2022.

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