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

NCT04043273 | Completed

• 1 other identifier: SATIS-Fab
• Study Type: observational
• Target: 69
• Locations: 1 country
• Sites: 15

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.

Conditions

Fabry Disease; Anderson Fabry Disease

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (1)

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

Interventions

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.

Whole cohort

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• \- 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)

Sponsors & Collaborators

• Amicus Therapeutics France SAS: lead

Study Sites (15)

CHU d'Angers

Angers, 49000, France

Location

CHU Pellegrin

Bordeaux, 33000, France

Location

Hôpital Côte de Nacre

Caen, 14033, France

Location

CHU de Dijon

Dijon, 21000, France

Location

CHRU de Lille

Lille, 59037, France

Location

Hôpital Femme Mère Enfant

Lyon, 69000, France

Location

Hôpital de la Conception

Marseille, 13000, France

Location

CHU de Nantes

Nantes, 44000, France

Location

Hopital Tenon

Paris, 75000, France

Location

Groupe Hospitalier Diaconesses Croix Saint-Simon

Paris, France

Location

CHU de Rennes

Rennes, 35000, France

Location

CHU de Rouen

Rouen, 76100, France

Location

CHU de Strasbourg

Strasbourg, 67000, France

Location

CHU de Toulouse

Toulouse, 31000, France

Location

CHU de Tours

Tours, 37000, France

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

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

Study Officials

• Olivier Lidove, MD

  Groupe Hospitalier Diaconesses Croix Saint-Simon

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2019
• First Posted: August 2, 2019
• Study Start: April 4, 2019
• Primary Completion: January 30, 2023
• Study Completion: January 30, 2023
• Last Updated: May 23, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (15)