Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
NCT ID: NCT04043273
Last Updated: 2024-05-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
69 participants
OBSERVATIONAL
2019-04-04
2023-01-30
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Whole cohort
The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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
16 Years
ALL
No
Sponsors
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Amicus Therapeutics France SAS
INDUSTRY
Responsible Party
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Principal Investigators
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Olivier Lidove, MD
Role: PRINCIPAL_INVESTIGATOR
Groupe Hospitalier Diaconesses Croix Saint-Simon
Locations
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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
Hopital Tenon
Paris, , France
Groupe Hospitalier Diaconesses Croix Saint-Simon
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, , France
Countries
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Other Identifiers
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SATIS-Fab
Identifier Type: -
Identifier Source: org_study_id
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