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Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis

This study has been completed.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
University of California, San Diego
Information provided by:
National Center for Research Resources (NCRR) Identifier:
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: December 2003

OBJECTIVES: I. Establish a computerized databank to monitor the progress of patients with cystinosis treated with cysteamine.

II. Track and monitor all patients including renal transplant, dialysis and post renal transplants.


Study Type: Observational
Study Design: Primary Purpose: Screening

Resource links provided by NLM:

Further study details as provided by National Center for Research Resources (NCRR):

Estimated Enrollment: 400
Study Start Date: October 1999
Detailed Description:


Data are collected from patients and physicians. Information includes disease, treatment, family history, demographic, and physical exam data. The dates of kidney transplantation and/or dialysis initiation are also recorded.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


Nephropathic cystinosis diagnosis by white-cell cystine measurement, including infantile and late-onset forms

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Please refer to this study by its identifier: NCT00004312

United States, California
University of California San Diego School of Medicine
La Jolla, California, United States, 92093-0652
Sponsors and Collaborators
National Center for Research Resources (NCRR)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
University of California, San Diego
Study Chair: Jerry A. Schneider University of California, San Diego
  More Information Identifier: NCT00004312     History of Changes
Other Study ID Numbers: NCRR-M01RR00827-1196
Study First Received: October 18, 1999
Last Updated: June 23, 2005

Keywords provided by National Center for Research Resources (NCRR):
rare disease
renal and genitourinary disorders

Additional relevant MeSH terms:
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases processed this record on May 22, 2017