Clinical trial NCT03321604: Kidney Information Network for Disease Research and Education
Official title: Kidney Information Network for Disease Research and Education (KINDRED)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: ESRD
Fabry Disease
Rare Diseases
Kidney Diseases
End Stage Renal Disease
Genetic Diseases, X-Linked
Keywords: Hemodialysis
Kidney
Dialysis
Kindred
Fabry
Massachusetts General Hospital
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Genzyme, a Sanofi Company
name: Scott and White Hospital & Clinic
First posted: 2017-10-25
Enrollment: 338
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Adults receiving dialysis

Brief summary: In this study, Investigators will conduct a prospective cohort study of dialysis patients by collecting research-quality information on patient characteristics, comorbid diseases and laboratory markers used in routine practice, as well as novel biochemical markers and genetic data. Investigators will utilize data from the cohort to test the independent relationship between biochemical and genetic markers and Fabry disease and other rare diseases.

Detailed description: KINDRED is a prospective observational cohort study that will enroll up to 5,000 dialysis patients throughout the United States with and without known diagnosis of Fabry disease but at risk for Fabry disease (e.g., unknown cause of renal disease, young male, initiating dialysis without a kidney biopsy, etc).This tissue repository will allow for genetic screening of a geographically diverse population, assist in identification of rare diseases in ESRD, and collect research-quality information on risk factors and outcomes from an ethnically heterogeneous population of dialysis patients. In this study, Investigators will use a novel method of performing research in this population by leveraging the internet to enhance and accelerate recruitment of potential subjects to participate in a genetic screening/tissue repository protocol.

Eligibility criteria: Inclusion Criteria:

* Adults between 18 and 65 with ESRD undergoing dialysis.

Exclusion Criteria:

* Kidney biopsy unlikely to be Fabry disease; those with a diagnosis that is clinically or biochemically proven not to be Fabry disease.

Primary outcomes:
measure: Genetically determined Fabry disease prevalence amongst a heterogenous patient population with end-stage renal disease on dialysis in the US not previously known to have Fabry disease.
timeFrame: 5 years
Secondary outcomes:
measure: Differences in patient characteristics of dialysis patients diagnosed with Fabry disease vs. dialysis patients who do not have Fabry disease.
timeFrame: 5 years
measure: Differences in patient characteristics of individuals diagnosed with Fabry disease initiating dialysis after 2013 vs. patients with Fabry disease who started dialysis before 2003.
timeFrame: 5 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
References: