Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
Status
Conditions
Treatments
Details and patient eligibility
About
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.
Full description
Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study.
Enrollment
Sex
Ages
Volunteers
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)
Trial design
69 participants in 1 patient group
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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