NCT03145779Withdrawn
Evaluation of Phenotypic Variability in Fabry Disease
Source: ClinicalTrials.gov · Last updated 3 Dec 2020
Reason given: Funding
Brief summary (registry, in English)
Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels. Understanding how the genotype influences the phenotype or clinical presentation can help us understand which patients are at risk for the complications of Fabry disease. This study aims to follow the natural history of this disease will help us understand and predict long-term outcomes for patients.
- Status (registry)
- Withdrawn
- Study type
- Observational
- Enrollment
- 0 (actual)
- First submitted
- 3 May 2017
- Start date
- Jul 2020
- Primary completion date
- Jul 2030
- Lead sponsor
- Boston Children's Hospital
- Diseases
- Fabry disease
- NCT number
- NCT03145779
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- No medical records available
- No record of genotype
- Not capable of providing informed consent
- Currently involved in any clinical trial
Who the study is looking for
- Individuals who carry a classic alpha-galactosidase gene (GLA) mutation
- All ages
- Medical records available including previous genetic testing.
- Capable of providing informed consent with assent for patients less than 18 years
- Not currently involved in any other clinical trials.
Age in the registry: 1 year to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Boston Children's Hospital | Boston | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.