Search / Trial NCT00001491

Analysis of the Nervous System in Patients With Fabry's Disease

Launched by NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE (NINDS) · Nov 3, 1999

Trial Information

Current as of December 14, 2024

Completed

Keywords

Ceramidetrihexosidase Deficiency Sural Nerve Biopsy Nerve Conduction Studies Quantitative Sensory Testing Pgp 9.5 Levels (Skin, Serum And Csf) Fabry Disease Fabrys Fabry's

ClinConnect Summary

OBJECTIVE: The purpose of this protocol is to study the natural history of Fabry disease in the different organ systems, understand its pathogenesis, and develop adequate clinical outcome measures for therapy trials. Development of sensitive outcome measures is a prerequisite of enzyme or gene replacement trials in patients with Fabry's disease. STUDY POPULATION: Patients with Fabry disease of all ages. The patients are not foregoing available treatment to participate in this protocol. Experimental treatment is not part of this study. STUDY DESIGN: Patients will typically be seen once a yea...

Gender

ALL

Eligibility criteria

  • * INCLUSION CRITERIA:
  • All patients with Fabry's disease will be considered as potential candidates for this study.
  • EXCLUSION CRITERIA:
  • All candidates must be serologically nonreactive for human immunodeficiency (AIDS) virus. HIV positive patients will be excluded because of the effects of the latter illness on the nervous system.
  • Patients with Fabry's disease will be excluded from participation if they have additional illnesses such as cancer, diabetes or vasculitis that could potentially involve the nervous system.
  • The general health and well being of each candidate must be sufficient to allow for a modest amount of blood drawing, collection of appropriate laboratory specimens and performance of necessary roentgenograpic and magnetic resonance (MR) imaging studies. In addition, each candidate must be able to return to the National Institutes of Health (NIH) annually for monitoring of clinical and laboratory parameters.

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

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