Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences

In the shared decision-making process, patients should express their expectations and preferences regarding treatment to the physician. A specific questionnaire addressing needs and expectations of Fabry patients has been built at the initiative of Amicus. In addition, this questionnaire also evaluates the benefit of treatment from the patient's perspective. Nothing is known until now on patient's expectations, potential clustering of patients regarding their expectations and evaluation of treatment benefit from the patients perspective.

Study objectives are differentiated according to the study phase (inclusion and follow-up). At inclusion, the primary objective is to cluster patients according to their needs and expectations regarding treatment. During follow-up, the primary objective is to evaluate treatment benefit in relation with patients needs and expectations.

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Key information

Conditions

Fabry Disease Anderson Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

16 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

CHU d'Angers, Angers, France

About this study

Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patients ≥ 16 years old
Diagnosed with Fabry disease
With amenable mutation
Decision by clinician to start or pursue ongoing ERT or migalastat
Non-opposition form to participate in the study signed

Exclusion criteria

Concomitant patient participation in an interventional clinical study (Category 1 interventional research or category 2 interventional research according to the Jardé law classification in France)

Treatment and study plan

Noninterventional characterization of patients expectations and preferences regarding their treatment

Behavioral

Patients will be clustered before and after a principal components analysis (PCA) on Patients Needs Questionnaire (PNQ). Observations will be clustered by the Ward method. The number of clusters will be chosen using the Kolinski criteria. Characteristics of patients will be described by cluster.

Primary outcomes

Homogeneous clusters of patients according to their needs and expectations towards the treatment received.

Time frame: Baseline

Number of clusters will be defined using a non-supervised classification method.
Treatment benefit, derived from Patients Benefit Index (PBI), in relation to patients needs and expectations towards their specific treatment.

Time frame: 12 month

Patients Benefit Index (PBI): descriptive statistics. The PBI will be calculated by reference to the PNQ measured at the preceding visit

The proportion of patients with PBI ≥ 1 will be described with a two-sided 95% confidence interval.

Sponsors and collaborators

Lead sponsor

Amicus Therapeutics France SAS

Industry

Registry information

Official study title

Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (SATIS-Fab)

Important dates

Study start: 2019
Primary completion: 2023
Study completion: 2023
First posted: Aug 2, 2019
Registry last updated: May 23, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed