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D000795: Fabry Disease

Developed by Shray Alag, The Harker School
Sections: Correlations, Clinical Trials, and HPO

Correlations computed by analyzing all clinical trials.

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Correlated Drug Terms (0)


Name (Synonyms) Correlation

Correlated MeSH Terms (0)


Name (Synonyms) Correlation

Correlated HPO Terms (1)


Name (Synonyms) Correlation
HP:0001071 Angiokeratoma corporis diffusum HPO 1.00

Clinical Trials

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There is one clinical trial.


1 Fabry Outcome Survey (FOS)

The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease. The data from FOS may provide guidance to healthcare professionals about disease treatment options.

NCT03289065
Conditions
  1. Fabry Disease
MeSH:Fabry Disease
HPO:Angiokeratoma corporis diffusum

Primary Outcomes

Description: An adverse event (AE) is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or ADR that meets one or more of the following criteria/outcomes is classified as SAE whether considered to be related to the pharmaceutical product or not: death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.

Measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time: Baseline to year 20

Description: An infusion-related reaction (IRR) is defined as an AE that has been assessed as at least possibly related to treatment with Replagal and occurs during an infusion or up to 24 hours post Replagal infusion.

Measure: Number of Participants With Infusion-related Reactions (IRRs)

Time: Baseline to year 20

Description: Renal function will be measured by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for adults and by Counahan-Barratt for children (<18 years).

Measure: Renal Function by Estimated Glomerular Filtration Rate (eGFR)

Time: Baseline to year 20

Description: Left ventricular mass index (LVMI) will be assessed from baseline or from birth (age at event) to evaluate the course of Fabry disease in participants who are currently untreated or are being treated with an approved Fabry treatment.

Measure: Left Ventricular Mass Index (LVMI)

Time: Baseline to year 20

Description: Age at mortality event (survival) will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in Fabry Outcome Survey (FOS)

Measure: Age at Mortality Event (survival)

Time: Baseline to year 20

Description: Time to first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.

Measure: Time to First Morbidity Event

Time: Baseline to year 20

Description: Age at first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.

Measure: Age at First Morbidity Event

Time: Baseline to year 20

HPO Nodes


HPO

Alphabetical listing of all HPO terms. Navigate: Correlations   Clinical Trials

Reports

Data processed on September 26, 2020.

An HTML report was created for each of the unique drugs, MeSH, and HPO terms associated with COVID-19 clinical trials. Each report contains a list of either the drug, the MeSH terms, or the HPO terms. All of the terms in a category are displayed on the left-hand side of the report to enable easy navigation, and the reports contain a list of correlated drugs, MeSH, and HPO terms. Further, all reports contain the details of the clinical trials in which the term is referenced. Every clinical trial report shows the mapped HPO and MeSH terms, which are also hyperlinked. Related HPO terms, with their associated genes, protein mutations, and SNPs are also referenced in the report.

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