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

Completed

• Conditions: Fabry Disease; Anderson Fabry Disease
• Interventions: Behavioral: Noninterventional characterization of patients expectations and preferences regarding their treatment

• Registration Number: NCT04043273
• Lead Sponsor: Amicus Therapeutics France SAS

Brief 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.

Detailed Description

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

Study Timeline

• Study Start: 2019-04-04
• Study First Submitted: 2019-06-28
• Study First Submitted QC: 2019-07-31
• Study First Posted: 2019-08-02
• Primary Completion: 2023-01-30
• Study Completion: 2023-01-30
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-22
• Last Update Posted: 2024-05-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 69

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)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Whole cohort	Noninterventional characterization of patients expectations and preferences regarding their treatment	The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment

Primary Outcome Measures

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

Trial Locations

Locations (15)

CHU d'Angers; 🇫🇷 Angers, France

Hôpital Côte de Nacre; 🇫🇷 Caen, France

CHU de Dijon; 🇫🇷 Dijon, France

CHRU de Lille; 🇫🇷 Lille, France

Hopital Tenon; 🇫🇷 Paris, France

Groupe Hospitalier Diaconesses Croix Saint-Simon; 🇫🇷 Paris, France

CHU de Rennes; 🇫🇷 Rennes, France

CHU de Rouen; 🇫🇷 Rouen, France

Hôpital Femme Mère Enfant; 🇫🇷 Lyon, France

Hôpital de la Conception; 🇫🇷 Marseille, France

CHU de Strasbourg; 🇫🇷 Strasbourg, France

CHU de Toulouse; 🇫🇷 Toulouse, France

CHU Pellegrin; 🇫🇷 Bordeaux, France

CHU de Nantes; 🇫🇷 Nantes, France

CHU de Tours; 🇫🇷 Tours, France