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

Sponsor

Amicus Therapeutics France SAS (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04043273

Collaborator

(none)

Enrollment

Locations

47.1

Anticipated Duration (Months)

4.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Fabry Disease Anderson Fabry Disease	Behavioral: Characterization of patients expectations and preferences regarding their treatment

Detailed Description

Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study. Decision to treat the patient should be made independently of the patient's participation in the study.

Study Design

Study Type:

Observational

Actual Enrollment :

69 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (SATIS-Fab)

Actual Study Start Date :

Mar 1, 2019

Anticipated Primary Completion Date :

Nov 1, 2022

Anticipated Study Completion Date :

Feb 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Whole cohort The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment	Behavioral: Characterization of 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.

Arm

Intervention/Treatment

Whole cohort

The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment

Behavioral: Characterization of 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.

Outcome Measures

Primary Outcome Measures

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.
Evaluate treatment benefit 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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

16 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy 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)

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	CHU Angers	Angers	France	49000
2	CHU Pellegrin	Bordeaux	France	33000
3	Hôpital Côte de Nacre	Caen	France	14033
4	CHU Dijon	Dijon	France	21000
5	CHRU de Lille	Lille	France	59037
6	Hôpital Femme Mère Enfant	Lyon	France	69000
7	Hôpital de la Conception	Marseille	France	13000
8	CHU Nantes	Nantes	France	44000
9	Tenon	Paris	France	75000
10	Groupe hospitalier Diaconesses Croix Saint-Simon	Paris	France
11	CHU Rennes	Rennes	France	35000
12	CHU Rouen	Rouen	France	76100
13	CHU Strasbourg	Strasbourg	France	67000
14	CHU Toulouse	Toulouse	France	31000
15	CHU Tours	Tours	France	37000

Sponsors and Collaborators

Amicus Therapeutics France SAS

Investigators

Principal Investigator: Olivier Lidove, MD, Groupe Hospitalier Diaconesses Croix Saint-Simon

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Amicus Therapeutics France SAS

ClinicalTrials.gov Identifier:

NCT04043273

Other Study ID Numbers:

SATIS-Fab

First Posted:

Aug 2, 2019

Last Update Posted:

Jul 27, 2022

Last Verified:

Jul 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Fabry Disease

Study Results

No Results Posted as of Jul 27, 2022