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

Completed

Conditions studied: Gaucher Disease

In brief

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.

Key facts

Study ID

NCT01136304

Run by

University Research Foundation for Lysosomal Storage Diseases, Inc.

People needed

173

Starts

2010-04-01

Expected to finish

2013-12-01

Last updated by the study team

2015-06-01

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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

Where it is running

• Tower Cancer Research Foundation — Beverly Hills, California, United States
• Northwest Oncology Hematology Associates PA — Coral Springs, Florida, United States

Full record on ClinicalTrials.gov

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