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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04043273
• Other study ID #: SATIS-Fab
• Status: Completed
• Start date: April 4, 2019
• Est. completion date: January 30, 2023

Study information

• Verified date: May 2024
• Source: Amicus Therapeutics France SAS
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 69
• Est. completion date: January 30, 2023
• Est. primary completion date: January 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Anderson Fabry Disease
• Fabry Disease

Intervention

Behavioral:
• Noninterventional characterization of patients expectations and preferences regarding their treatment
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.

Locations

Country	Name	City	State
France	CHU d'Angers	Angers
France	CHU Pellegrin	Bordeaux
France	Hôpital Côte de Nacre	Caen
France	CHU de Dijon	Dijon
France	CHRU de Lille	Lille
France	Hôpital Femme Mère Enfant	Lyon
France	Hôpital de la Conception	Marseille
France	CHU de Nantes	Nantes
France	Groupe Hospitalier Diaconesses Croix Saint-Simon	Paris
France	Hopital Tenon	Paris
France	CHU de Rennes	Rennes
France	CHU de Rouen	Rouen
France	CHU de Strasbourg	Strasbourg
France	CHU de Toulouse	Toulouse
France	CHU de Tours	Tours

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics France SAS

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Homogeneous clusters of patients according to their needs and expectations towards the treatment received.	Number of clusters will be defined using a non-supervised classification method.	Baseline
Primary	Treatment benefit, derived from Patients Benefit Index (PBI), in relation to patients needs and expectations towards their specific treatment.	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.	12 month