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Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 173

Last updated 2015-06-01

No results posted yet for this study

Summary

With the participation of an international consortium of investigators, the investigators will evaluate the validity of a new severity score system called DS3 for adult patients with Gaucher disease. The investigators hypothesize that initial DS3 scores will be predictive of both disease progression and patterns of response including imiglucerase dose sensitivity and completeness and maintenance of response and that sequential DS3 scores will accurately portray either clinical progression of disease or improvement in response to treatment. The investigators will also collect DNA specimens that in future research will be used in conjunction with the DS3 scores to evaluate determinants of the clinical course and the response to treatments for Gaucher disease.

Conditions

  • Gaucher Disease

Interventions

DRUG

Imiglucerase

Imiglucerase intravenous infusions regardless of dose or schedule of administration.

Sponsors & Collaborators

  • University of Pittsburgh

    collaborator OTHER

  • University Research Foundation for Lysosomal Storage Diseases, Inc.

    lead OTHER

Principal Investigators

  • Neal J Weinreb, MD · University Research Foundation for Lysosomal Storage Diseases, Inc.

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-04-30
Primary Completion
2013-12-31
Completion
2013-12-31

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 NCT01136304 on ClinicalTrials.gov