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Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

2014-08-27 03:57:09 | BioPortfolio

Summary

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.

Description

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).

Study Design

Primary Purpose: Treatment

Conditions

Cystinosis

Intervention

cysteamine hydrochloride

Status

Completed

Source

FDA Office of Orphan Products Development

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:57:09-0400

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PubMed Articles [223 Associated PubMed Articles listed on BioPortfolio]

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Medical and Biotech [MESH] Definitions

A radiation-protective agent that oxidizes in air to form CYSTAMINE. It can be given intravenously or orally to treat radiation sickness. The bitartrate has been used for the oral treatment of nephropathic cystinosis.

A metabolic disease characterized by the defective transport of CYSTINE across the lysosomal membrane due to mutation of a membrane protein cystinosin. This results in cystine accumulation and crystallization in the cells causing widespread tissue damage. In the KIDNEY, nephropathic cystinosis is a common cause of RENAL FANCONI SYNDROME.

Ethylamines, including CYSTEAMINE, that contain a sulfhydryl group in their structure.

A pharmaceutical preparation of sitagliptin phosphate and metformin hydrochloride that is used in the treatment of TYPE 2 DIABETES.

Compounds and drugs that react with CYSTINE and convert it into a compound that can be more easily metabolized or intracellularly transported. Drugs in this class have been used to treat CYSTINOSIS.

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