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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01019629
Other study ID # 008-230
Secondary ID
Status Completed
Phase N/A
First received November 23, 2009
Last updated February 12, 2018
Start date January 29, 2009
Est. completion date December 7, 2017

Study information

Verified date February 2018
Source Baylor Research Institute
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

To determine if patients with a deficiency of alpha-galactosidase A are at-risk for cardiac complications that commonly occur in the general population


Description:

Fabry disease is an X-linked deficiency of alpha-galactosidase A resulting primarily in an accumulation of globotriaosylceramide (Gb3) in virtually all organs and systems. The main complications of Fabry disease are a 20-fold increased risk of ischemic stroke, cardiac disease including cardiomyopathy, atrio-ventricular conduction defects, a wide variety of arrhythmias, valvular dysfunction (insufficiency or stenosis) and cardiac vascular disease as well as progressive renal failure. Fabry disease cannot be easily diagnosed in patients with routine EKGs, echocardiograms or MRIs. Screening non-selected at-risk populations of patients with ischemic stroke or cardiac disease for urinary Gb3, alpha-galactosidase A activity and GLA gene mutations should enable the identification of patients previously undiagnosed with Fabry disease among the general population of patients with heart disease and stroke


Recruitment information / eligibility

Status Completed
Enrollment 2724
Est. completion date December 7, 2017
Est. primary completion date April 17, 2013
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria:

- Any diagnosis of heart disease.

- Male or Female

- Able to donate 12 cc of whole blood and 10 cc of urine

Exclusion Criteria:

- No diagnosis of cardiac disease.

- Unable to donate 12 cc of whole blood and/or 10 cc of urine

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United States Baylor Institute of Metabolic Disease Dallas Texas

Sponsors (1)

Lead Sponsor Collaborator
Baylor Research Institute

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Identify GLA gene variants Collect blood and urine sample one time only for analysis Once
See also
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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
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Completed NCT01947634 - Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. N/A
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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
Recruiting NCT06007768 - Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
Recruiting NCT05698901 - Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
Active, not 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
Active, not recruiting NCT03566017 - Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease Phase 3
Recruiting NCT06065605 - Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease