Covid 19 Research using Clinical Trials (Home Page)
HP:0001071: Angiokeratoma corporis diffusumHPO
Developed by Shray Alag
Clinical Trial MeSH HPO Drug Gene SNP Protein Mutation
Correlated Drug Terms (0)
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Correlated HPO Terms (0)
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There is one clinical trial.
Clinical Trials
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 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
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 20Description: 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 20Description: 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 20Description: 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 20Description: 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 20Description: 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 20Description: 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