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Sponsors and Collaborators: |
FDA Office of Orphan Products Development Sigma Tau Pharmaceuticals, Inc. |
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Information provided by: | FDA Office of Orphan Products Development |
ClinicalTrials.gov Identifier: | NCT00010426 |
OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation.
II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.
Condition | Intervention |
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Cystinosis |
Drug: cysteamine hydrochloride |
Study Type: | Interventional |
Study Design: | Treatment |
Estimated Enrollment: | 30 |
Study Start Date: | December 1999 |
Estimated Study Completion Date: | February 2001 |
PROTOCOL OUTLINE: This is a randomized, multicenter study Patients are randomized to receive the current formulation of cysteamine hydrochloride as drops in one eye and the new formulation of cysteamine hydrochloride as drops in the other eye.
Patients receive the two formulations of cysteamine hydrochloride in their assigned eyes every hour during waking hours daily for 6 months (safety study) or for 1 year (efficacy study).
Ages Eligible for Study: | 1 Year to 50 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
--Prior/Concurrent Therapy--
--Patient Characteristics--
Study ID Numbers: | 199/15704, SIGMATAU-FDR001769 |
Study First Received: | February 2, 2001 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00010426 |
Health Authority: | United States: Federal Government |
cystinosis rare disease renal and genitourinary disorders |
Cystinosis Metabolism, Inborn Errors Cysteamine Metabolic Diseases |
Genetic Diseases, Inborn Lysosomal Storage Diseases Rare Diseases Metabolic disorder |
Radiation-Protective Agents Physiological Effects of Drugs Protective Agents Pharmacologic Actions |