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

NCT03683966 | Unknown

• 1 other identifier: AT-IIP-2018
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Rare Diseases; Fabry Disease; Adherence, Medication; Quality of Life

Keywords

Fabry; Chaperone therapy; Migalastat; Pharmacological adherence

Outcome Measures

Primary Outcomes (1)

• Adherence to oral therapy with migalastat according to Medication Assessment Questionnaire

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

  From date of inclusion up to104 weeks (2 years)

Secondary Outcomes (1)

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

  From date of inclusion up to104 weeks (2 years)

Other Outcomes (1)

• Pain according to SF-36 and Wuerzburg pain questionnaire

  From date of inclusion up to104 weeks (2 years)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

• No informed consent
• Withdrawal of informed consent

Sponsors & Collaborators

• Wuerzburg University Hospital: lead
• Charite University, Berlin, Germany: collaborator
• University Hospital Muenster: collaborator
• Health Care Center Dr. Markus Cybulla, Muellheim: collaborator

Study Sites (1)

Wuerzburg University Hospital

Würzburg, Bavaria, 97080, Germany

RECRUITING

Related Publications (7)

• 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.

  PMID: 29452394 BACKGROUND

• 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.

  PMID: 29018006 BACKGROUND

• 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.

  PMID: 27509102 BACKGROUND

• 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.

  PMID: 27834756 BACKGROUND

• 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.

  PMID: 27657681 BACKGROUND

• 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.

  PMID: 32198894 BACKGROUND

• 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.

  PMID: 30506669 BACKGROUND

MeSH Terms

Conditions

Rare Diseases; Fabry Disease; Medication Adherence

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Patient Compliance; Patient Acceptance of Health Care; Treatment Adherence and Compliance; Health Behavior; Behavior

Study Officials

• Peter Nordbeck, MD, PhD

  Wuerzburg University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Peter Nordbeck, MD, PhD

CONTACT

004993120139181; nordbeck_p@ukw.de

Jonas Muentze, MD

CONTACT

004993120139958; muentze_j@ukw.de

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head Consultant Cardiology and Chief of Electrophysiology

Study Record Dates

• First Submitted: September 3, 2018
• First Posted: September 25, 2018
• Study Start: October 27, 2017
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2023
• Last Updated: July 11, 2022

Record last verified: 2022-07

Locations

DE (1)