Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | 18 - 65 |
| Updated: | 4/2/2016 |
| Start Date: | November 1999 |
OBJECTIVES:
I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with
Gaucher disease.
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.
Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on
clinical severity of disease and response to therapy.
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
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University of Pittsburgh The University of Pittsburgh is a state-related research university, founded as the...
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