Search / Trial NCT00001416

Bone Response to Enzyme Replacement in Gaucher's Disease

Launched by NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE (NINDS) · Dec 9, 2002

Trial Information

Current as of December 05, 2024

Completed

Keywords

Bone Disease Calcitriol Enzyme Replacement Therapy Gaucher's Disease Vitamin D

ClinConnect Summary

The purpose of this study is to examine the response of the skeleton to repeated infusions of macrophage-targeted glucocerebrosidase (CEREDASE (Trademark) ) in splenectomized patients with type I Gaucher's disease. The magnitude and rate of development of the skeletal response will be monitored non-invasively. Theoretically, an enhanced response should occur in patients supplemented with pharmacologic doses of 1, 25-dihydroxyvitamin D3 (calcitriol), since calcium absorption and enzyme delivery to bone marrow macrophages should be increased in this setting. These issues will be addressed in ...

Gender

ALL

Eligibility criteria

  • Splenectomized Gaucher patients.
  • Aged 18-45 who have not received enzyme therapy for at least 1 year.
  • No patients with other illnesses (pulmonary, liver, kidney, bone, hematologic).

About National Institute Of Neurological Disorders And Stroke (Ninds)

The National Institute of Neurological Disorders and Stroke (NINDS) is a leading component of the National Institutes of Health (NIH), dedicated to advancing our understanding of the brain and nervous system through innovative research. NINDS sponsors and conducts clinical trials aimed at developing effective treatments and improving patient outcomes for a wide range of neurological disorders, including stroke, epilepsy, multiple sclerosis, and neurodegenerative diseases. By fostering collaboration among researchers, healthcare providers, and patient communities, NINDS plays a pivotal role in transforming scientific discoveries into clinical applications, ultimately enhancing the quality of life for individuals affected by neurological conditions.

Locations

Bethesda, Maryland, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Discussion 0

Similar Trials