Fabry DiseaseNCT04043273
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
An observational study of Fabry Disease and Anderson Fabry Disease, run by Amicus Therapeutics France SAS at 15 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Fabry Disease, Anderson Fabry Disease
- What is being tested?
- Noninterventional characterization of patients expectations and preferences regarding their treatment
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 69 participants
- Where
- 15 sitesFrance
- Study dates
- 4 Apr 2019to 30 Jan 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Fabry disease with the potential to be treated either by ERT or migalastat in real-life conditions and according to the SmPC
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
15 sites in 1 country.
15 sites in 1 country
France15 sites
CHU d'Angers
Angers, 49000
CHU Pellegrin
Bordeaux, 33000
Hôpital Côte de Nacre
Caen, 14033
CHU de Dijon
Dijon, 21000
CHRU de Lille
Lille, 59037
Hôpital Femme Mère Enfant
Lyon, 69000
Hôpital de la Conception
Marseille, 13000
CHU de Nantes
Nantes, 44000
Hopital Tenon
Paris, 75000
Groupe Hospitalier Diaconesses Croix Saint-Simon
Paris
CHU de Rennes
Rennes, 35000
CHU de Rouen
Rouen, 76100
CHU de Strasbourg
Strasbourg, 67000
CHU de Toulouse
Toulouse, 31000
CHU de Tours
Tours, 37000
About this study
Official title: 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.
Read the detailed description
Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study.
- Conditions
- Fabry Disease, Anderson Fabry Disease
- Being tested
- Noninterventional characterization of patients expectations and preferences regarding their treatment
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Noninterventional characterization of patients expectations and preferences regarding their treatmentBehavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| Whole cohort | The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment Receives: Behavioral: Noninterventional characterization of patients expectations and preferences regarding their treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Homogeneous clusters of patients according to their needs and expectations towards the treatment received.
Number of clusters will be defined using a non-supervised classification method.
Measured: Baseline
Treatment benefit, derived from Patients Benefit Index (PBI), in relation to patients needs and expectations towards their specific treatment.
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.
Measured: 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2019
- Study start4 Apr 2019
- Primary completion30 Jan 2023
- Study completion30 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics France SAS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olivier Lidove, MD, Principal investigator, Groupe Hospitalier Diaconesses Croix Saint-Simon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04043273
- Sponsor's protocol ID
- SATIS-Fab
- Status last verified
- May 2024
- First submitted
- 28 Jun 2019
- Last updated
- 23 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lidove O, Masseau A, Pugnet G, Lacombe D, Dussol B, Bekri S, Hagege A, Martinez C, Fardini Y, Fouilhoux A, Noel E. Treatment-related benefit and satisfaction in patients with Fabry disease in France: insight into patients' expectations and preferences from the prospective, non-interventional SATIS-Fab study. Orphanet J Rare Dis. 2026 Apr 1;21(1):162. doi: 10.1186/s13023-026-04285-7.
PubMed 41923254
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (SATIS-Fab)
- ClinicalTrials.gov ID
- NCT04043273
- Acronym / sponsor ID
- SATIS-Fab
- Conditions
- Fabry Disease, Anderson Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Amicus Therapeutics France SASIndustry (pharma, biotech, devices)
- Study start
- 4 Apr 2019 (actual)
- Primary completion
- 30 Jan 2023 (actual)
- Study completion
- 30 Jan 2023 (actual)
- First posted
- 2 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Whole cohort | – | Behavioral: Noninterventional characterization of patients expectations and preferences regarding their treatmentThe whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Fabry disease with the potential to be treated either by ERT or migalastat in real-life conditions and according to the SmPC
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Noninterventional characterization of patients expectations and preferences regarding their treatmentPatients 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. | Behavioural | Whole cohort |
Primary outcome
Homogeneous clusters of patients according to their needs and expectations towards the treatment received.
Time frame: 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.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2024.
- Derived from this study: PubMed 41923254Lidove O, Masseau A, Pugnet G, Lacombe D, Dussol B, Bekri S, Hagege A, Martinez C, Fardini Y, Fouilhoux A, Noel E. Treatment-related benefit and satisfaction in patients with Fabry disease in France: insight into patients' expectations and preferences from the prospective, non-interventional SATIS-Fab study. Orphanet J Rare Dis. 2026 Apr 1;21(1):162. doi: 10.1186/s13023-026-04285-7.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Olivier Lidove, MD principal investigator
Groupe Hospitalier Diaconesses Croix Saint-Simon
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease and Anderson Fabry Disease, run by Amicus Therapeutics France SAS at 15 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Homogeneous clusters of patients according to their needs and expectations towards the treatment received. (time frame: Baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04043273, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2024.