Status:
COMPLETED
Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
Lead Sponsor:
University Research Foundation for Lysosomal Storage Diseases, Inc.
Collaborating Sponsors:
University of Pittsburgh
Conditions:
Gaucher Disease
Eligibility:
All Genders
18+ years
Brief 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. ...
Detailed Description
GD1 is a prototypical lysosomal storage disorder and the first disorder to have compelling evidence of successful treatment with enzyme replacement therapy. The common clinical manifestations are hema...
Eligibility Criteria
Inclusion
- Adult patients with Type 1 Gaucher disease regardless of treatment status who are enrolled in the International Collaborative Gaucher Group (ICCG) Gaucher Registry and who are cared for at one of the participating research sites.
Exclusion
- Children under the age of 18 years
- Patients with Type 3 Gaucher disease
- Patients who have declined to be enrolled in the ICCG Gaucher Registry
- Patients not cared for at one of the participating research sites
Key Trial Info
Start Date :
April 1 2010
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 1 2013
Estimated Enrollment :
173 Patients enrolled
Trial Details
Trial ID
NCT01136304
Start Date
April 1 2010
End Date
December 1 2013
Last Update
June 1 2015
Active Locations (2)
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1
Tower Cancer Research Foundation
Beverly Hills, California, United States, 90211
2
Northwest Oncology Hematology Associates PA
Coral Springs, Florida, United States, 33065