OBJECTIVES: I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.

Condition	Treatment or Intervention	Phase
Gaucher's Disease	Drug: glucocerebrosidase	Phase II

Further Study Details: 

Expected Total Enrollment:  24

PROTOCOL OUTLINE: Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.

Ages Eligible for Study:  18 Years   -   65 Years,  Genders Eligible for Study:  Both

Criteria

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay At least 3 organ systems affected, based on the following criteria: Anemia Thrombocytopenia Organomegaly Bone deterioration on radiograph Pulmonary compromise Symptoms compromise daily activities or risk longevity No neurologic disease

Pennsylvania
      University of Pittsburgh, Pittsburgh,  Pennsylvania,  15260,  United States; Recruiting

Study chairs or principal investigators

John Barranger,  Study Chair,  University of Pittsburgh   

Study ID Numbers:  199/11725; UPITTS-M1230
Record last reviewed:  December 2003
Last Updated:  October 13, 2004
Record first received:  October 18, 1999
ClinicalTrials.gov Identifier:  NCT00004293
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08