Anderson Fabry Disease, Fabry Disease
Conditions
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.
Detailed description
Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study.
Interventions
BEHAVIORALNoninterventional 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
Amicus Therapeutics France SAS
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Homogeneous clusters of patients according to their needs and expectations towards the treatment received. | Baseline | Number of clusters will be defined using a non-supervised classification method. |
| Treatment benefit, derived from Patients Benefit Index (PBI), in relation to patients needs and expectations towards their specific treatment. | 12 month | 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. |
Countries
France
Outcome results
None listed