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NCT04043273
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
trial testing Noninterventional characterization of patients expectations and preferences regarding their treatment in Fabry Disease in 69 participants. Completed in 30 January 2023.
30 January 2023
Quick facts
| Lead sponsor | Amicus Therapeutics France SAS |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 69 |
| Start date | 4 April 2019 |
| Primary completion | 30 January 2023 |
| Estimated completion | 30 January 2023 |
| Sites | 15 locations across France |
Drugs / interventions tested
- Noninterventional characterization of patients expectations and preferences regarding their treatment
Conditions studied
- Fabry Disease — all drugs for Fabry Disease →
- Anderson Fabry Disease — all drugs for Anderson Fabry Disease →
Sponsor
Amicus Therapeutics France SAS
Who can join
16 and older, any sex, with Fabry Disease or Anderson Fabry Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
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.
Lidove O, Masseau A, Pugnet G, Lacombe D, et al · · 2026 · PMID 41923254 · DOI 10.1186/s13023-026-04285-7
Verify or expand the search:
- PubMed search for NCT04043273
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Fabry Disease
Currently open trials in the same condition.
- NCT07187440 — A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease · recruiting
- NCT06776419 — the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease · recruiting
- NCT06539624 — Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease · NA · recruiting
- NCT07277361 — Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment · recruiting
- NCT06270316 — Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease · Phase 1, PHASE2 · recruiting
Other Amicus Therapeutics France SAS trials
Trials by the same sponsor.
- NCT04602364 — French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04043273 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Amicus Therapeutics France SAS
- Last refreshed: 23 May 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04043273.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing