Status:
COMPLETED
Effect of Migalastat on Cardiac Involvement in Fabry Disease
Lead Sponsor:
Ospedale San Donato
Collaborating Sponsors:
Amicus Therapeutics
Institute of Biomedicine and Molecular Immunology - CNR
Conditions:
Fabry Disease
Heart Diseases
Eligibility:
All Genders
16+ years
Brief Summary
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 sma...
Detailed Description
Anderson-Fabry disease is a rare X-linked lysosomal disorder, mainly affecting cardiovascular, renal and nervous systems. Cardiac injury is the leading cause of death in these patients. Heart involvem...
Eligibility Criteria
Inclusion
- Genetic diagnosis of Fabry Disease and amenable mutation
- Clinical indication to Migalastat
- Signs of clinical or preclinical cardiac involvement (low T1 values with or without left ventricular hypertrophy)
- Age \>16
- Ability to give a complete informed consent (for minor patients informed consent will be given by parents)
Exclusion
- Contraindication to Migalastat (pregnancy, age \<16, Glomerular Filtration Rate \<30 ml/min, hypersensitivity to the active ingredient)
- Contraindication to CMR study (metallic fragment or foreign body, known claustrophobia, PaceMaker/Implantable Cardioverter Defibrillator not CMR conditional, electronic implant or device, eg, insulin pump or other infusion pump)
Key Trial Info
Start Date :
January 10 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
January 22 2021
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT03838237
Start Date
January 10 2018
End Date
January 22 2021
Last Update
March 18 2021
Active Locations (1)
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1
IRCCS Policlinico San Donato
San Donato Milanese, Milano, Italy, 20097