Clinical trial NCT04043273: Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
Official title: Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (SATIS-Fab)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Fabry Disease
Anderson Fabry Disease
Lead sponsor: Amicus Therapeutics France SAS
Sponsor class: INDUSTRY
First posted: 2019-08-02
Enrollment: 69
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Fabry disease with the potential to be treated either by ERT or migalastat in real-life conditions and according to the SmPC

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Homogeneous clusters of patients according to their needs and expectations towards the treatment received.
timeFrame: Baseline
measure: Treatment benefit, derived from Patients Benefit Index (PBI), in relation to patients needs and expectations towards their specific treatment.
timeFrame: 12 month
Secondary outcomes:
Other outcomes:
Locations:
city: Angers
country: France
facility: CHU d'Angers
city: Bordeaux
country: France
facility: CHU Pellegrin
city: Caen
country: France
facility: Hôpital Côte de Nacre
city: Dijon
country: France
facility: CHU de Dijon
city: Lille
country: France
facility: CHRU de Lille
city: Lyon
country: France
facility: Hôpital Femme Mère Enfant
city: Marseille
country: France
facility: Hôpital de la Conception
city: Nantes
country: France
facility: CHU de Nantes
city: Paris
country: France
facility: Hopital Tenon
city: Paris
country: France
facility: Groupe Hospitalier Diaconesses Croix Saint-Simon
city: Rennes
country: France
facility: CHU de Rennes
city: Rouen
country: France
facility: CHU de Rouen
city: Strasbourg
country: France
facility: CHU de Strasbourg
city: Toulouse
country: France
facility: CHU de Toulouse
city: Tours
country: France
facility: CHU de Tours
Study officials:
affiliation: Groupe Hospitalier Diaconesses Croix Saint-Simon
name: Olivier Lidove, MD
role: PRINCIPAL_INVESTIGATOR
References: