Krabbe Disease Global Patient Registry
ConditionKrabbe Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorState University of New York at Buffalo
About this trial
The purpose of this study is to develop a clinical database of individuals diagnosed with Krabbe disease in order to determine which symptoms herald the onset of clinical disease in the various phenotypes of Krabbe disease; to determine whether level of GALC enzyme activity, or a specific genetic mutation predict the clinical course; and to determine which neurodiagnostic tests predict onset and/or severity of the disease.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Anyone diagnosed with Krabbe disease
Anyone at-risk for Krabbe disease
Family members of someone diagnosed with, or at-risk for, Krabbe disease.
Disqualifiers
Anyone who is not diagnosed with, or at-risk for, Krabbe disease
Anyone who is not a family member of someone diagnosed with, or at-risk for, Krabbe disease
Trial population
This study seeks enrollment by anyone of any age or gender who has been diagnosed with Krabbe disease, and also: * Anyone at-risk for Krabbe disease * Family members of someone diagnosed with, or at-risk for, Krabbe disease. This may consist of adults unable to consent; individuals who are not yet adults; and pregnant women.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Overall Survival
The longevity of participants will be recorded using their date of death, or conclusion of this study, whichever occurs first.
Time frame
up to 5 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
State University of New York at Buffalo
Lead sponsor
Rare Diseases Clinical Research Network
Collaborator
National Center for Advancing Translational Sciences (NCATS)
Collaborator
Lysosomal Disease Network
Collaborator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator
Source ClinicalTrials.gov