Fabry Disease Clinical Trials

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FunctiOnal caPaciTy Evaluation Using cardIopulMonary Testing and Stress echocArdiography in Anderson-Fabry Disease Patients: OPTIMA-FD Study

Status: Recruiting
Location: See all (5) locations...
Study Type: Observational
SUMMARY

The goal of this observational study is to observe the relation between excercise parameters - assessed by CPET - and rest/stress hemodynamic parameters - assessed by echocardiogram and CMR - in patients with a genetic diagnosis of Anderson-Fabry Disease. Participants will undergo: * baseline evaluation: clinical evaluation, disease staging with FASTEX and MSSI, KCCQ for quality of life assessment, resting 12-leads ECG, 6MWT, CPET-ESE and contrast-enhanced CMR; * before 36 months from baseline: resting 12-leads ECG, 2D rest and stress echocardiogram, CPET-ESE, contrast-enhanced CMR, disease staging with FASTEX and MSSI and KCCQ for quality of life assessment; * up to 7 years from baseline: clinical follow-up.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Patients with a genetic diagnosis of AFD, according to current guidelines;

• Informed written consent with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care (for patients age \<18 years old, written consent from a caregiver is mandatory).

Locations
Other Locations
Italy
Spedali Civili Hospital
ACTIVE_NOT_RECRUITING
Brescia
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
ACTIVE_NOT_RECRUITING
Milan
Fondazione IRCCS San Gerardo dei Tintori
ACTIVE_NOT_RECRUITING
Monza
IRCCS Policlinico San Donato
RECRUITING
San Donato Milanese
Regina Margherita Hospital
ACTIVE_NOT_RECRUITING
Turin
Contact Information
Primary
Massimo Piepoli, MD, PhD
massimo.piepoli@grupposandonato.it
+39 0252774942
Backup
Gianluigi Guida, MD
gianluigi.guida@grupposandonato.it
+39 0252774942
Time Frame
Start Date: 2024-11-13
Estimated Completion Date: 2032-01
Participants
Target number of participants: 100
Treatments
Normal cardiac parameters and normal T1
AFD patients with normal cardiac parameters and normal T1.
Normal cardiac parameters and reduced T1
AFD patients with no LVH and myocardial reduced T1 .
LVH without LGE
Patients with LVH without LGE.
Advanced cardiomyopathy with LVH and LGE
AFD patients with advanced cardiomyopathy with LVH and LGE.
Related Therapeutic Areas
Sponsors
Leads: IRCCS Policlinico S. Donato

This content was sourced from clinicaltrials.gov