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 May 11, 2025
Completed
Keywords
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.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Bethesda, Maryland, United States
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
Similar Trials