MedPath

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Phase 2
Conditions
Gaucher's Disease
Registration Number
NCT00004293
Lead Sponsor
National Center for Research Resources (NCRR)
Brief Summary

OBJECTIVES:

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

Detailed Description

PROTOCOL OUTLINE:

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

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
24
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

University of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

© Copyright 2025. All Rights Reserved by MedPath