Overview

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Status:
Unknown status
Trial end date:
1969-12-31
Target enrollment:
0
Participant gender:
All
Summary
OBJECTIVES: I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
National Center for Research Resources (NCRR)
Collaborator:
University of Pittsburgh
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