PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

Launched by NATIONAL INSTITUTE OF MENTAL HEALTH (NIMH) · Nov 3, 1999

Keywords

ClinConnect Summary

The purpose of this clinical study is to evaluate the biochemical and therapeutic effects and safety of enzyme replacement therapy using polyethyleneglycol (PEG) modified glucocerebrosidase for the treatment of Gaucher disease and to evaluate the benefit to risk ratio. The study is designed to determine the safety and efficacy in Gaucher patients of recombinantly produced human glucocerebrosidase, which is PEG modified. Parameters to be monitored include hemoglobin, platelet counts, organ size, and extent of bony involvement. Pharmacokinetic, pharmacodynamic, and antibody studies will also ...

Gender

ALL

Eligibility criteria

• • Patients must be at least 3 years of age.
• • Must have a biochemically confirmed (enzyme) and/or genetically confirmed diagnosis of type 1 or type 3 Gaucher disease.
• • Clinical or laboratory signs suggesting need for therapy which will include at least 2 of the following: hemoglobin less than 11 gm/dl; platelets less than 90,000/mm(3); hepatomegaly and/or splenomegaly.
• • Patient/Guardian must provide written informed consent.
• • No pregnant or breast feeding women.
• • No women/men of reproductive potential unless they agree to use an effective contraceptive method.
• • No patients treated with alglucerase or imiglucerase during the 6 months prior to study entry.
• • No patients with the diagnosis of type 2 Gaucher disease.
• • No patients who have a life-threatening disease or are gravely ill.
• • No patients who have rapidly progressing fatal illness or concomitant malignancy.
• • No patients who have a chronic infectious disease including HIV or hepatitis B.
• • No patients chronically on other medications which may interfere with the drug's metabolism or activity.
• • No patients who received blood transfusion within a month prior to study entry.