NCT00455104 Canadian Fabry Disease Initiative (CFDI) National Registry
| NCT ID | NCT00455104 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Canadian Fabry Research Consortium |
| Condition | Fabry Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 600 participants |
| Start Date | 2007-01 |
| Primary Completion | 2029-10 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 600 participants in total. It began in 2007-01 with a primary completion date of 2029-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
CFDI NATIONAL REGISTRY Fabry disease is a rare, inherited, genetic condition due to a deficiency of an enzyme called alpha-galactosidase A. This enzyme deficiency causes the small blood vessels to accumulate a substance called glycolipid. Without sufficient levels of the enzyme, alpha-galactosidase A, persons with Fabry Disease develop severe neuropathic pain, kidney disease, heart disease, stroke and/or premature death; often before the age of 60. Fabry Disease is estimated to affect approximately one out of every 40,000 males and up to twice as many females in Canada. We do not have the exact number of persons in Canada who have this disease. A common problem in studying rare conditions is the difficulty in identifying the majority of people suffering from such a disease. Gathering their health information in order to better understand the natural disease progression and its response to treatment is difficult. Early ERT studies involving humans had small numbers of subjects and the studies were of short duration. The results of these clinical studies did lead to approval of the therapy in many countries around the world including Canada. To date though, evidence of the usefulness of ERT and its direct impact on the natural course of Fabry disease has been limited, while its cost continues to be very high. As a result of these issues, there will need to be continued and long-term collection of information related to the effectiveness of ERT and other treatments to better document its true clinical outcomes in Canadian people with Fabry disease. The Canadian Fabry Disease Initiative National Registry (CFDI-NR) is an observational, voluntary registry designed to collect outcomes data on Fabry disease from people living in Canada.
Eligibility 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
Contact & Investigator
Michael L West, MD
PRINCIPAL INVESTIGATOR
Queen Elizabeth II Health Sciences Centre (Capital District Health Authority), Halifax, Nova Scotia, Canada
Frequently Asked Questions
Who can join the NCT00455104 clinical trial?
This trial is open to participants of all sexes, aged 5 Years or older, up to 85 Years, studying Fabry Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT00455104 currently recruiting?
Yes, NCT00455104 is actively recruiting participants. Contact the research team at mlwest@dal.ca for enrollment information.
Where is the NCT00455104 trial being conducted?
This trial is being conducted at Calgary, Canada, Vancouver, Canada, Halifax, Canada, Toronto, Canada and 1 additional location.
Who is sponsoring the NCT00455104 clinical trial?
NCT00455104 is sponsored by Canadian Fabry Research Consortium. The principal investigator is Michael L West, MD at Queen Elizabeth II Health Sciences Centre (Capital District Health Authority), Halifax, Nova Scotia, Canada. The trial plans to enroll 600 participants.