A Phase I and II Study of PEG-Glucocerebrosidase in Patients With Type 1 or Type 3 Gaucher Disease
Lysodase
Brain Diseases+14
+ Brain Diseases, Metabolic
+ Central Nervous System Diseases
Treatment Study
Summary
Study start date: October 1, 1993
Actual date on which the first participant was enrolled.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 be evaluated.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.18 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
National Institute of Mental Health (NIMH)
Bethesda, United StatesOpen National Institute of Mental Health (NIMH) in Google Maps