Trials / Completed
CompletedNCT04043273
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (SATIS-Fab)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 69 (actual)
- Sponsor
- Amicus Therapeutics France SAS · Industry
- Sex
- All
- Age
- 16 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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. |
Timeline
- Start date
- 2019-04-04
- Primary completion
- 2023-01-30
- Completion
- 2023-01-30
- First posted
- 2019-08-02
- Last updated
- 2024-05-23
Locations
15 sites across 1 country: France
Source: ClinicalTrials.gov record NCT04043273. Inclusion in this directory is not an endorsement.