National registry for Fabry disease in Canada
Canadian Fabry Disease Initiative National Registry: Outcomes of Rare Disease Therapeutics and Cardiovascular Risk Factor Modification
This study is setting up a national registry to collect health information from people with Fabry disease in Canada to better understand how the disease progresses and how treatments work.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 600 (estimated) |
| Ages | 5 Years to 85 Years |
| Sex | All |
| Sponsor | Canadian Fabry Research Consortium Research network |
| Locations | 5 sites (Calgary, Alberta and 4 other locations) |
| Trial ID | NCT00455104 on ClinicalTrials.gov |
What this trial studies
The Canadian Fabry Disease Initiative (CFDI) National Registry aims to establish a comprehensive registry to collect health information from individuals diagnosed with Fabry disease across Canada. This observational study will gather data on clinical outcomes, treatment responses, and potential biomarkers related to the disease. Participants will undergo regular assessments, including medical history, physical exams, and various diagnostic tests, to better understand the disease's progression and treatment efficacy. The registry will help address the challenges of studying this rare condition by identifying and monitoring affected individuals.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 5 to 85 years with a clinical diagnosis of Fabry disease who can provide informed consent.
Not a fit: Patients who may not benefit from this study include those with non-disease causing mutations or an estimated life expectancy of less than 12 months.
Why it matters
Potential benefit: If successful, this registry could enhance understanding of Fabry disease and improve patient management and treatment outcomes.
How similar studies have performed: Other studies have successfully utilized national registries to improve understanding and management of rare diseases, making this approach promising.
Eligibility criteria
Show full inclusion / exclusion criteria
INCLUSION CRITERIA: * Age 5 years and older, up to \& including age 85 years; and * Able to give informed consent; and * A clinical diagnosis of Fabry disease; and * Compliance with all the clinic visits, interviews and assessments during the study period; and * A Canadian citizen or a landed immigrant EXCLUSION CRITERIA: * Inability to give informed consent; or * Problem complying with all the clinic visits, interviews and assessments during the study period; or * An estimated life expectancy of less than 12 months * Under 5 years of age * Non-disease causing mutation
Where this trial is running
Calgary, Alberta and 4 other locations
- Alberta Children's Hospital — Calgary, Alberta, Canada (Recruiting)
- Vancouver General Hospital Adult Metabolic Diseases Clinic — Vancouver, British Columbia, Canada (Recruiting)
- Queen Elizabeth II Health Sciences Centre — Halifax, Nova Scotia, Canada (Recruiting)
- Toronto Western Hospital — Toronto, Ontario, Canada (Recruiting)
- University of Montreal, Department of Medicine — Montreal, Quebec, Canada (Recruiting)
Study contacts
- Principal investigator: Michael L West, MD — Queen Elizabeth II Health Sciences Centre (Capital District Health Authority), Halifax, Nova Scotia, Canada
- Study coordinator: Michael L. West, MD
- Email: mlwest@dal.ca
- Phone: 902-473-4023
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.