Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

National Center for Research Resources (NCRR)24 enrolled

Overview

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

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

Study Type

INTERVENTIONAL

Purpose

TREATMENT

Enrollment

Conditions

Gaucher's Disease

Interventions

glucocerebrosidaseDRUG

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

RECRUITING

Data from ClinicalTrials.gov

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