Bone Response to Enzyme Replacement in Gaucher's Disease
Completed
National Institute of Neurological Disorders and Stroke (NINDS)
Phase 2
1993-12-01
The purpose of this study is to examine how the skeleton responds to repeated doses of enzyme
replacement therapy in patients with type I Gaucher's disease who have had their spleens
removed.
Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation
in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in
adults but occurs most severely in infants, in whom cerebroside also accumulates in neurons.
Patients with Gaucher's disease experience enlargement of the liver and spleen and bone
destruction. The condition is passed from generation to generation through autosomal
recessive inheritance.
Type I is the most common form. It is a chronic non-neuronopathic form, meaning the disease
does not affect nerve cells. The symptoms of type I can appear at any age.
In this study patients will be divided into three groups. Each group will receive different
doses of enzyme replacement (Ceredase). In addition, two of the three groups will also
receive doses of a form of vitamin D (calcitriol). Researchers believe the groups receiving
vitamin D will have an improved response as compared to those patients only receiving enzyme
replacement.
Patients in each group who respond to enzyme replacement with increases in bone density will
be compared to the other treatment groups.
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