- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04043273
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)
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
-
-
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Angers, France, 49000
- CHU Angers
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Bordeaux, France, 33000
- CHU Pellegrin
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Caen, France, 14033
- Hopital Cote de Nacre
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Dijon, France, 21000
- CHU Dijon
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Lille, France, 59037
- CHRU de Lille
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Lyon, France, 69000
- Hôpital Femme Mère Enfant
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Marseille, France, 13000
- Hôpital de la Conception
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Nantes, France, 44000
- CHU Nantes
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Paris, France
- Groupe Hospitalier Diaconesses Croix Saint-Simon
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Paris, France, 75000
- Tenon
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Rennes, France, 35000
- CHU Rennes
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Rouen, France, 76100
- CHU Rouen
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Strasbourg, France, 67000
- CHU Strasbourg
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Toulouse, France, 31000
- CHU Toulouse
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Tours, France, 37000
- CHU Tours
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
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)
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Whole cohort
The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment
|
Patients will be clustered before and after a principal components analysis (PCA) on 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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Homogeneous clusters of patients according to their needs and expectations towards the treatment received.
Time Frame: Baseline
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Number of clusters will be defined using a non-supervised classification method.
|
Baseline
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Evaluate treatment benefit in relation to patients needs and expectations towards their specific treatment.
Time Frame: 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. |
12 month
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Olivier Lidove, MD, Groupe Hospitalier Diaconesses Croix Saint-Simon
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ANTICIPATED)
Study Completion (ANTICIPATED)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Metabolic Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Cerebral Small Vessel Diseases
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Fabry Disease
Other Study ID Numbers
- SATIS-Fab
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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