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NCT03838237: MAIORA
Effect of Migalastat on Cardiac Involvement in Fabry Disease
trial testing Cardiological evaluation in Fabry Disease in 18 participants. Completed in 22 January 2021.
22 January 2021
Quick facts
| Lead sponsor | Ospedale San Donato |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 18 |
| Start date | 10 January 2018 |
| Primary completion | 22 January 2021 |
| Estimated completion | 22 January 2021 |
| Sites | 1 location across Italy |
Drugs / interventions tested
- Cardiological evaluation
Conditions studied
- Fabry Disease — all drugs for Fabry Disease →
- Heart Diseases — all drugs for Heart Diseases →
Sponsor
Ospedale San Donato — full company profile →
Who can join
16 and older, any sex, with Fabry Disease or Heart Diseases. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Anderson-Fabry Disease (AFD) is one of the rare lysosomal storage disorders for which a cause - specific therapy is available. Recently, a new specific drug has been marketed, namely Migalastat, a small-molecule pharmacological chaperone. The effect of Migalastat on cardiac involvement has been assessed so far by 2D echocardiography, demonstrating a significant reduction in left ventricular (LV) mass after 18 months of therapy. Calculation of LV mass by 2D echocardiography is limited by geometrical assumptions and quality of echocardiographic window, with a strong impact on accuracy. Cardiac Magnetic Resonance (CMR) overcomes these limitations, thus representing the gold standard technique for ventricular mass, volumes and function estimation. Moreover, CMR offers the unique possibility to perform a non-invasive tissue characterization, including the detection of both myocardial fibrosis by Late Gadolinium Enhancement and sphingolipid storage by T1 mapping. Beyond an accurate morphological description and a detailed tissue characterization, a complete cardiological assessment should also integrate functional data and bio-humoral profile. This study is designed to provide a comprehensive evaluation of the therapeutic effect of Migalastat (123 mg every other day) on cardiac involvement after 18 months of therapy, integrating a morphological, functional and bio-humoral assessment.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
2021 TSOC Expert Consensus on the Clinical Features, Diagnosis, and Clinical Management of Cardiac Manifestations of Fabry Disease.
Hung CL, Wu YW, Lin CC, Lai CH, et al · · 2021 · cited 29× · PMID 34257484 · DOI 10.6515/acs.202107_37(4).20210601a -
Effect of Migalastat on cArdiac InvOlvement in FabRry DiseAse: MAIORA study.
Camporeale A, Bandera F, Pieroni M, Pieruzzi F, et al · · 2023 · cited 22× · PMID 36669872 · DOI 10.1136/jmg-2022-108768
Verify or expand the search:
- PubMed search for NCT03838237
- 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 Ospedale San Donato trials
Trials by the same sponsor.
- NCT07368816 — OEOd-Based Wound Dressing in Post-Surgical Diabetic Foot Ulcers · NA · completed
- NCT04995640 — CILCA Arch Registry: Management and Outcomes of Open and Endovascular Repair · unknown
- NCT04594889 — Comparison of Sirolimus vs Paclitaxel Drug Eluting Balloon for Below-the-knee Angioplasty in Critical Limb Ischemia · NA · unknown
- NCT03824626 — Biomechanical Reappraisal of Planning for Thoracic Endovascular Aortic Repair · unknown
- NCT03493971 — Hemodynamics and Autonomic and Cognitive Performance After Carotid Revascularization Procedures · NA · 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 NCT03838237 (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 Ospedale San Donato
- Last refreshed: 18 March 2021
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/NCT03838237.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing