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Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 4

Last updated 2015-07-28

No results posted yet for this study

Summary

The purpose of this study are: to characterize and understand the natural history of disease progression in WD and CESD, and to provide historical controls for WD and CESD for developing clinical treatment trials. The hypothesis is that the variability and clinical progression in WD and CESD is large and represents a continuum of severities from a lethal infantile to near normal adults with only "fatty livers".

Conditions

  • Wolman Disease
  • Cholesterol Ester Storage Disease
  • Acid Cholesteryl Ester Hydrolase Deficiency, Type 2

Interventions

OTHER

There are no interventions in this study.

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    collaborator NIH

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Gregory A Grabowski, MD · Children's Hospital Medical Center, Cincinnati

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-11-30
Primary Completion
2014-05-31
Completion
2014-05-31

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 NCT01884220 on ClinicalTrials.gov