Clinical Trials Logo

Clinical Trial Summary

Fabry disease is an inherited disorder that affects many organs in the body, including the heart. Men and women are both affected, with average life expectancy reduced by 10-20 years. The heart muscle can become thick and scarred in over half of patients, eventually resulting in heart failure, abnormal heart rhythm and death. We are focused on improving the detection of early heart disease before irreversible damage has occurred in order to improve patient outcomes. The techniques that have traditionally been used to look for disease in the heart are inadequate, as they often identify abnormalities once permanent scarring has already developed. Patients with heart damage have poor outcomes even with treatment. Therefore, it is critical to develop an earlier and reliable way to identify subtle disease in the heart, so that treatment can be initiated early when patients will benefit the most. We have previously shown that cardiac magnetic resonance imaging (MRI) can identify changes in the heart muscle in patients with Fabry disease. We hypothesize that new cardiac MRI techniques called T1 and T2 mapping will improve our ability to detect early abnormalities in the heart. Early detection of cardiac disease may enable a personalized treatment approach, potentially improving patient outcomes. The results of this study will help identify which patients might benefit from early initiation of treatment to prevent bad outcomes in the future by using cardiac MRI to identify those at higher risk.

Clinical Trial Description


Study Design

Related Conditions & MeSH terms

NCT number NCT04856059
Study type Interventional
Source University Health Network, Toronto
Contact Kate Hanneman, MD
Phone 416-323-6400
Email [email protected]
Status Not yet recruiting
Phase N/A
Start date May 17, 2021
Completion date December 31, 2026

See also
  Status Clinical Trial Phase
Recruiting NCT04455230 - A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 N/A
Completed NCT01218659 - Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease Phase 3
Completed NCT00304512 - A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease Phase 2
Not yet recruiting NCT04189601 - Complement Activation in the Lysosomal Storage Disorders
Not yet recruiting NCT03228940 - Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease Phase 1/Phase 2
Active, not recruiting NCT03180840 - Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients Phase 3
Recruiting NCT02994303 - Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy N/A
Completed NCT01947634 - Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. N/A
Recruiting NCT01695161 - Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. N/A
Completed NCT01853852 - A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects Phase 1
Completed NCT00701415 - A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms Phase 3
Completed NCT00068107 - Dosing Study of Replagal in Patients With Fabry Disease Phase 2
Completed NCT01997489 - Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. Phase 4
Enrolling by invitation NCT03566017 - Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease Phase 3
Recruiting NCT03305250 - Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease N/A
Terminated NCT00526071 - Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study Phase 2
Recruiting NCT04519749 - An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Classic Fabry Disease Phase 1/Phase 2
Not yet recruiting NCT04440254 - Long Duration Holter ECG in Fabry Disease N/A
Recruiting NCT04184986 - Screening of Fabry Disease in Patients With GI Symptoms
Recruiting NCT03683966 - MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study