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Krabbe Disease Global Patient Registry

NCT02993796 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 60

Last updated 2025-02-10

No results posted yet for this study

Summary

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

  • Krabbe Disease

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • Lysosomal Disease Network

    collaborator OTHER

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

    collaborator NIH

  • State University of New York at Buffalo

    lead OTHER

Principal Investigators

  • Thomas J. Langan, MD · Clinical Director, Clinical Research, Institute for Myelin and Glial Exploration

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-09-30
Primary Completion
2026-09-30
Completion
2026-09-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02993796 on ClinicalTrials.gov