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

NCT04043273 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 69

Last updated 2024-05-23

No results posted yet for this study

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

Interventions

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.

Sponsors & Collaborators

  • Amicus Therapeutics France SAS

    lead INDUSTRY

Principal Investigators

  • Olivier Lidove, MD · Groupe Hospitalier Diaconesses Croix Saint-Simon

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-04
Primary Completion
2023-01-30
Completion
2023-01-30

Countries

  • France

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04043273 on ClinicalTrials.gov