Actively Recruiting

All Genders
NCT02993796

Krabbe Disease Global Patient Registry

Led by State University of New York at Buffalo · Updated on 2025-02-10

60

Participants Needed

1

Research Sites

626 weeks

Total Duration

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Sponsors

S

State University of New York at Buffalo

Lead Sponsor

R

Rare Diseases Clinical Research Network

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Krabbe Disease Global Patient Registry

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Anyone diagnosed with Krabbe disease
  • Anyone at-risk for Krabbe disease
  • Family members of someone diagnosed with, or at-risk for, Krabbe disease
Not Eligible

You will not qualify if you...

  • 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

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

State University of New York at Buffalo

Buffalo, New York, United States, 14203

Actively Recruiting

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Research Team

T

Thomas J. Langan, MD

CONTACT

A

Amy Barczykowski

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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