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

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

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.

Study Overview

• Status: Completed
• Conditions: Fabry Disease; Anderson Fabry Disease
• Intervention / Treatment: Behavioral: Noninterventional characterization of patients expectations and preferences regarding their treatment

Detailed Description

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

• Study Type: Observational
• Enrollment (Actual): 69

Contacts and Locations

Study Locations

• France
  • Angers, France, 49000
    • CHU d'Angers
  • Bordeaux, France, 33000
    • CHU Pellegrin
  • Caen, France, 14033
    • Hôpital Côte De Nacre
  • Dijon, France, 21000
    • CHU de Dijon
  • Lille, France, 59037
    • CHRU de Lille
  • Lyon, France, 69000
    • Hôpital Femme Mère Enfant
  • Marseille, France, 13000
    • Hopital de la Conception
  • Nantes, France, 44000
    • CHU de Nantes
  • Paris, France
    • Groupe Hospitalier Diaconesses Croix Saint-Simon
  • Paris, France, 75000
    • Hôpital Tenon
  • Rennes, France, 35000
    • CHU de Rennes
  • Rouen, France, 76100
    • CHU de Rouen
  • Strasbourg, France, 67000
    • CHU de Strasbourg
  • Toulouse, France, 31000
    • CHU de Toulouse
  • Tours, France, 37000
    • Chu De Tours

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Whole cohort; The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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

Collaborators and Investigators

• Sponsor: Amicus Therapeutics France SAS
• Investigators: Principal Investigator: Olivier Lidove, MD, Groupe Hospitalier Diaconesses Croix Saint-Simon

Study record dates

Study Major Dates

• Study Start (Actual): April 4, 2019
• Primary Completion (Actual): January 30, 2023
• Study Completion (Actual): January 30, 2023

Study Registration Dates

• First Submitted: June 28, 2019
• First Submitted That Met QC Criteria: July 31, 2019
• First Posted (Actual): August 2, 2019

Study Record Updates

• Last Update Posted (Actual): May 23, 2024
• Last Update Submitted That Met QC Criteria: May 22, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Fabry Disease
• Other Study ID Numbers: SATIS-Fab

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No